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149 results for AAV

AAV Delivery System - [In Vivo] [AAV tropism, Delivery Systems Initiative] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice Evolving High Potency AAV Vectors for Neuromuscular Genome Editing name --> AAV --> AAV tropism --> AAV --> AAV --> AAV3b-ZsGreen-Cre AAVrh8-ZsGreen-Cre AAV9-CMV-Cre AAV6-ZsGreen-Cre AAV4-ZsGreen-Cre --> AAV name --> AAV --> AAV Tropism project See vector details Show Experiments (2) AAV Tropism project Cre Recombinase dose escalation study in Ai9 mice
AAV Tropism project Experiment - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice name --> AAV Tropism project --> AAV tropism --> AAV --> AAV Tropism project --> AAV6-ZsGreen-Cre AAV3b-ZsGreen-Cre AAV4-ZsGreen-Cre AAV8-ZsGreen-Cre AAV5-ZsGreen-Cre --> Ten AAV serotypes delivering Cre recombinase were tested by intravenous delivery into Ai9 mice and chacterized --> AAV name --> AAV Tropism project --> AAV Tropism project Lutz Cathleen M , Gao Guang-Ping , Heaney Jason D , Murray Stephen A , Lagor William Raymond , Dickinson Mary E Last Updated Date: 2023-02-10 Ten AAV serotypes delivering Cre recombinase were tested by intravenous delivery into Ai9 mice and chacterized for biodistribution across 20 tissues by quantitative PCR and imaging Show Project SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice
Conklin_Fast-Seq AAV Protocol Protocol Matched on: --> Conklin_Fast-Seq AAV Protocol --> Published procedure for AAV composition measurement using fast-seq. Maynard et al. name --> Conklin_Fast-Seq AAV Protocol Published procedure for AAV composition measurement using fast-seq. Maynard et al. Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings. Hum Gene Ther Methods . 2019 Dec;30(6):195-205. doi: 10.1089/hgtb.2019.110.
Heaney-SATC_Gao-Validation_Intratracheal Delivery of AAV in Mice Protocol - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> Heaney-SATC_Gao-Validation_Intratracheal Delivery of AAV in Mice --> Procedure for Intratracheal (IT) delivery of AAV in mouse lung. --> AAV name --> Heaney-SATC_Gao-Validation_Intratracheal Delivery of AAV in Mice Procedure for Intratracheal (IT) delivery of AAV in mouse lung. Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice Project [AAV tropism] Matched on: --> AAV tropism --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice name --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice Lutz Cathleen M , Gao Guang-Ping , Heaney Jason D , Murray Stephen A , Lagor William Raymond , Dickinson Mary E Last Updated Date: 2023-02-10 Show Experiments (1) AAV Tropism project
Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Project [Delivery Systems Initiative] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> Recombinant adeno-associated viruses (AAV) have emerged as safe and effective vectors for clinical gene The current proposal is on a comprehensive and innovative approach to evolve high potency AAV variants name --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Asokan Aravind Last Updated Date: 2020-11-19 Recombinant adeno-associated viruses (AAV) have emerged as safe and effective vectors for clinical gene therapy applications including systemic treatment of neuromuscular diseases such as Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD), and Giant Axonal Neuropathy (GAN) amongst others. However, genome editing in neuromuscular tissue, in particular, is challenging. The current proposal is on a comprehensive and innovative approach to evolve high potency AAV variants for systemic neuromuscular genome editing. Show Experiments (7) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Cre Recombinase dose escalation study in Ai9 mice Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
[Validation] Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Experiment - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular --> AAVcc47-Cre AAVcc47-SaCas9-Ai9 --> Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. --> AAV name --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Heaney Jason D Last Updated Date: 2021-03-30 Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues. Show Project BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Asokan Validation (Asokan, Aravind)
On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV --> AAV-Pcsk9 --> AAV was assessed one week after systemic administration of BE-eVLPs or AAV-Pcsk9 to C57BL/6 mice. name --> compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV --> compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV Chaikof Elliot L. Last Updated Date: 2022-04-15 On-target editing compared to off-target editing at 14 CIRCLE seq nominated sites in livers of an adenine base editor delivered by engineered virus-like particles (BE-eVLPs). Treated mice vs. untreated vs. AAV was assessed one week after systemic administration of BE-eVLPs or AAV-Pcsk9 to C57BL/6 mice. DNA sequencing reads containing A-T to G-C mutations within protospacer positions 4-10. Show Project Delivery Technologies for In Vivo Genome Editing
AAVS1_site_12 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_12 --> AAVS1_site_12 name --> AAVS1_site_12 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_13 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_13 --> AAVS1_site_13 name --> AAVS1_site_13 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_04 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_04 --> AAVS1_site_04 name --> AAVS1_site_04 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_11 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_11 --> AAVS1_site_11 name --> AAVS1_site_11 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_01 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_01 --> AAVS1_site_01 name --> AAVS1_site_01 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_05 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_05 --> AAVS1_site_05 name --> AAVS1_site_05 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_06 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_06 --> AAVS1_site_06 name --> AAVS1_site_06 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_08 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_08 --> AAVS1_site_08 name --> AAVS1_site_08 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_10 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_10 --> AAVS1_site_10 name --> AAVS1_site_10 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_03 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_03 --> AAVS1_site_03 name --> AAVS1_site_03 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_09 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_09 --> AAVS1_site_09 name --> AAVS1_site_09 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_02 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_02 --> AAVS1_site_02 name --> AAVS1_site_02 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_07 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_07 --> AAVS1_site_07 name --> AAVS1_site_07 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAVS1_site_14 Guide - [In Vitro] [Biological Systems] [Human] Matched on: name --> AAVS1_site_14 --> AAVS1_site_14 name --> AAVS1_site_14 --> AAVS1 Targets AAVS1 safe harbor locus Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
AAV6-ZsGreen-Cre Vector - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice --> AAV tropism --> AAV symbol --> AAV6-ZsGreen-Cre --> AAV6-ZsGreen-Cre --> AAV backbone with a bi-directional promoter driving zsGreen and Cre --> AAV --> AAV Tropism project AAV backbone with a bi-directional promoter driving zsGreen and Cre Show Experiments (1) AAV Tropism project
AAV9-Ai9-sgRNA2-CB-SaCas9 Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAV9-Ai9-sgRNA2-CB-SaCas9 --> AAV9-Ai9-sgRNA2-CB-SaCas9 AAV9-Ai9-sgRNA1-CB-SaCas9 --> AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus --> AAV AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
AAV9_pTR_self comp 2xU6-Ai9 guides Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAV9_pTR_self comp 2xU6-Ai9 guides --> AAV9_pTR_self comp 2xU6-Ai9 guides AAV9-CMV-SaCas9 --> AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting --> AAV AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
AAV9-ZsGreen-Cre Vector - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice --> AAV tropism --> AAV symbol --> AAV9-ZsGreen-Cre --> AAV9-ZsGreen-Cre --> AAV backbone with a bi-directional promoter driving zsGreen and Cre --> AAV --> AAV Tropism project AAV backbone with a bi-directional promoter driving zsGreen and Cre Show Experiments (1) AAV Tropism project
AAVcc47-CMV-SaCas9 Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAVcc47-CMV-SaCas9 --> AAVcc47-Ai9-sgRNA1 + sgRNA2 AAVcc47-CMV-SaCas9 --> AAV AAVcc47 delivering CMV driven SaCas9 Show Experiments (2) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
AAVcc47_pTR_self comp 2xU6-Ai9 guides Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAVcc47_pTR_self comp 2xU6-Ai9 guides --> AAV9-CMV-SaCas9 AAVcc47_pTR_self comp 2xU6-Ai9 guides --> AAV AAVcc47 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV7-ZsGreen-Cre Vector - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice --> AAV tropism --> AAV symbol --> AAV7-ZsGreen-Cre --> AAV7-ZsGreen-Cre --> AAV backbone with a bi-directional promoter driving zsGreen and Cre --> AAV --> AAV Tropism project AAV backbone with a bi-directional promoter driving zsGreen and Cre Show Experiments (1) AAV Tropism project
AAV8-ZsGreen-Cre Vector - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice --> AAV tropism --> AAV symbol --> AAV8-ZsGreen-Cre --> AAV8-ZsGreen-Cre --> AAV backbone with a bi-directional promoter driving zsGreen and Cre --> AAV --> AAV Tropism project AAV backbone with a bi-directional promoter driving zsGreen and Cre Show Experiments (1) AAV Tropism project
AAVcc47-Ai9-sgRNA1-CB-SaCas9 Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAVcc47-Ai9-sgRNA1-CB-SaCas9 --> AAVcc47-Ai9-sgRNA2-CB-SaCas9 AAVcc47-Ai9-sgRNA1-CB-SaCas9 --> AAV AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV-Pcsk9 Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-Pcsk9 --> AAV-Pcsk9 --> compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV Show Experiments (1) On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver
AAV9-Ai9-sgRNA1-CB-SaCas9 Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAV9-Ai9-sgRNA1-CB-SaCas9 --> AAV9-Ai9-sgRNA2-CB-SaCas9 AAV9-Ai9-sgRNA1-CB-SaCas9 --> AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus --> AAV AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
AAV9-CMV-SaCas9 Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAV9-CMV-SaCas9 --> AAV9_pTR_self comp 2xU6-Ai9 guides AAV9-Ai9-sgRNA1 + sgRNA2 AAV9-CMV-SaCas9 AAVcc47_pTR_self comp 2xU6-Ai9 guides --> AAV serotype 9 delivering CMV driven SaCas9 --> AAV AAV serotype 9 delivering CMV driven SaCas9 Show Experiments (3) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
AAVcc47-Cre Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: symbol --> AAVcc47-Cre --> AAVcc47-Cre --> AAV --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular AAV2/5 expressing Cre recombinase Show Experiments (1) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
AAVcc47-SaCas9-Ai9 Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: symbol --> AAVcc47-SaCas9-Ai9 --> AAVcc47-SaCas9-Ai9 --> AAV --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter Show Experiments (1) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
AAVcc84-GFP Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAVcc84-GFP --> AAVcc84-GFP --> AAV AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter Show Experiments (1) Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV5-ZsGreen-Cre Vector - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice --> AAV tropism --> AAV symbol --> AAV5-ZsGreen-Cre --> AAV5-ZsGreen-Cre --> AAV backbone with a bi-directional promoter driving zsGreen and Cre --> AAV --> AAV Tropism project AAV backbone with a bi-directional promoter driving zsGreen and Cre Show Experiments (1) AAV Tropism project
AAV9-Ai9-sgRNA1 + sgRNA2 Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAV9-Ai9-sgRNA1 + sgRNA2 --> AAV9-Ai9-sgRNA1 + sgRNA2 AAV9-CMV-SaCas9 --> AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus --> AAV AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus Show Experiments (2) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
AAVcc47-Ai9-sgRNA2-CB-SaCas9 Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAVcc47-Ai9-sgRNA2-CB-SaCas9 --> AAVcc47-Ai9-sgRNA2-CB-SaCas9 AAVcc47-Ai9-sgRNA1-CB-SaCas9 --> AAV AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
AAVcc47-CMV-Cre Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAVcc47-CMV-Cre --> AAVcc47-CMV-Cre --> AAV AAVcc47 delivering CMV Cre Recombinase Show Experiments (1) Cre Recombinase dose escalation study in Ai9 mice
AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAVrh10-ZsGreen-Cre Vector - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice --> AAV tropism --> AAV symbol --> AAVrh10-ZsGreen-Cre --> AAVrh10-ZsGreen-Cre --> AAV backbone with a bi-directional promoter driving zsGreen and Cre --> AAV --> AAV Tropism project AAV backbone with a bi-directional promoter driving zsGreen and Cre Show Experiments (1) AAV Tropism project
AAVrh74-ZsGreen-Cre Vector - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice --> AAV tropism --> AAV symbol --> AAVrh74-ZsGreen-Cre --> AAVrh74-ZsGreen-Cre --> AAV backbone with a bi-directional promoter driving zsGreen and Cre --> AAV --> AAV Tropism project AAV backbone with a bi-directional promoter driving zsGreen and Cre Show Experiments (1) AAV Tropism project
AAVrh8-ZsGreen-Cre Vector - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice --> AAV tropism --> AAV symbol --> AAVrh8-ZsGreen-Cre --> AAVrh8-ZsGreen-Cre --> AAV backbone with a bi-directional promoter driving zsGreen and Cre --> AAV --> AAV Tropism project AAV backbone with a bi-directional promoter driving zsGreen and Cre Show Experiments (1) AAV Tropism project
AAV4-ZsGreen-Cre Vector - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice --> AAV tropism --> AAV symbol --> AAV4-ZsGreen-Cre --> AAV4-ZsGreen-Cre --> AAV backbone with a bi-directional promoter driving zsGreen and Cre --> AAV --> AAV Tropism project AAV backbone with a bi-directional promoter driving zsGreen and Cre Show Experiments (1) AAV Tropism project
AAV9-GFP Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAV9-GFP --> AAV9-GFP --> AAV AAV2/9 self complementary vector expressing GFP driven by CBh promoter Show Experiments (1) Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
AAV9-mCherry Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAV9-mCherry --> AAV9-mCherry --> AAV AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter Show Experiments (1) Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
AAVcc81-GFP Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAVcc81-GFP --> AAVcc81-GFP --> AAV AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter Show Experiments (1) Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAVcc47-Ai9-sgRNA1 + sgRNA2 Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAVcc47-Ai9-sgRNA1 + sgRNA2 --> AAVcc47-Ai9-sgRNA1 + sgRNA2 AAVcc47-CMV-SaCas9 --> AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus --> AAV AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus Show Experiments (2) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
AAV9-CMV-Cre Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV symbol --> AAV9-CMV-Cre --> AAV9-CMV-Cre --> AAV serotype 9 delivering CMV Cre Recombinase --> AAV AAV serotype 9 delivering CMV Cre Recombinase Show Experiments (1) Cre Recombinase dose escalation study in Ai9 mice
AAVcc47-mCherry Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol --> AAVcc47-mCherry --> AAVcc47-mCherry --> AAV AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter Show Experiments (1) Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
AAV.pU1a-SpCas9 Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV.pU1a-SpCas9 --> AAV.pU1a-SpCas9 H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS Show Experiments (2) Testing AAV5 for activation of tdTomato in mouse airway Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
AAV3b-ZsGreen-Cre Vector - [In Vivo] [AAV tropism] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice --> AAV tropism --> AAV symbol --> AAV3b-ZsGreen-Cre --> AAV3b-ZsGreen-Cre --> AAV backbone with a bi-directional promoter driving zsGreen and Cre --> AAV --> AAV Tropism project AAV backbone with a bi-directional promoter driving zsGreen and Cre Show Experiments (1) AAV Tropism project
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 Vector - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: symbol --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 --> AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Tsai_T Cell Transfection Protocol Protocol - [In Vitro] [Biological Systems] [Human] Matched on: --> AAVS1_site_14 AAVS1_site_13 AAVS1_site_12 AAVS1_site_11 AAVS1_site_10 --> AAVS1 Procedure for T cell transfection. Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
demo VV03-Cre Vector [Mouse] Matched on: --> AAV DEMO viral vector for demo purpose
demo VV02-Cre Vector [Mouse] Matched on: --> AAV DEMO viral vector for demo purpose
Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV9-Ai9-sgRNA1 + sgRNA2 AAVcc47-Ai9-sgRNA1 + sgRNA2 AAVcc47-CMV-SaCas9 AAV9-CMV-SaCas9 --> single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV --> AAV name --> Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 Asokan Aravind Last Updated Date: 2021-09-16 A dual vector strategy was employed: one delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV cassettes). This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=5) by intravenous injection in Ai9 mice. A total dose of 4e12vg was injected into each mouse and vectors mixed in a 1:4 ratio of cas9 to guide RNA (1e12vg of CMV Sacas9 vector and 3e12vg of the sgRNA vector) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
[Validation] Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Experiment - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing --> AAV name --> Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to --> AAV BI28 (novel engineered variant) Heaney Jason D Last Updated Date: 2023-05-10 Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing. Editing detected and quantified in brain by generation of tdTomato fluorescent protein signal from Ai9 reporter mice Show Project BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV.pU1a-SpCas9 H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV name --> Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells Gao Guang-Ping Last Updated Date: 2021-09-21 AAV2/5 mediated gene editing in the mouse airway was tested by deliverying SpCas9 and guide RNAs targeting the Ai9 transgene in Ai9 transgenic mice. Gene editing quantified by tdTomato activation and cell specific markers for club and ciliated cell types. Show Project Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
SaCas9-Lagor Genome Editor - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV9-Ai9-sgRNA2-CB-SaCas9 AAV9-Ai9-sgRNA1 + sgRNA2 AAVcc47-CMV-SaCas9 AAV9-Ai9-sgRNA1-CB-SaCas9 AAV9-CMV-SaCas9 --> AAV Show Experiments (4) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV9-mCherry AAVcc47-mCherry --> AAV Asokan Aravind Last Updated Date: 2020-11-19 C57BL/6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding an mCherry reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification. Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Cre recombinase Genome Editor - [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] Matched on: --> AAVcc47-Cre --> Cre recombinase delivered by AAV (see vector details) --> AAV --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Cre recombinase delivered by AAV (see vector details) Show Experiments (3) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
A novel human T cell platform to define biological adverse effects of genome editing Experiment - [In Vitro] [Biological Systems] [Human] Matched on: --> AAVS1_site_14 AAVS1_site_13 AAVS1_site_12 AAVS1_site_11 AAVS1_site_10 --> AAVS1 Tsai Shengdar Q Last Updated Date: 2020-12-09 110 guide RNAs and SpCas9 were transfected into human T-cells. Indel rates were measured by targeted amplicon deep sequencing. Show Project A Novel Human T-Cell Platform to Define Biological Effects of Genome Editing
P3 Nucleofection Kit Delivery System - [In Vitro] [Biological Systems] [Human] Matched on: --> AAVS1_site_14 AAVS1_site_13 AAVS1_site_12 AAVS1_site_11 AAVS1_site_10 --> AAVS1 Nuceleofection kit to be used with Lonza's nucleofection system. Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
CD4/CD8 Human Primary T cell Model System - [In Vitro] [Biological Systems] [Human] Matched on: --> AAVS1_site_14 AAVS1_site_13 AAVS1_site_12 AAVS1_site_11 AAVS1_site_10 --> AAVS1 CD4/CD8 Human Primary T cell Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
SpCas9 Genome Editor - [In Vivo, In Vitro] [Delivery Systems Initiative, Biological Systems] [Human, Mouse] Matched on: --> AAVS1_site_14 AAVS1_site_13 AAVS1_site_12 AAVS1_site_11 AAVS1_site_10 --> AAV.pU1a-SpCas9 PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV --> AAVS1 HA-SV40NLS-SpCas9-SV40NLS Show Experiments (4) Testing AAV5 for activation of tdTomato in HEK293T cells Testing AAV5 for activation of tdTomato in mouse airway A novel human T cell platform to define biological adverse effects of genome editing Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV9-Ai9-sgRNA2-CB-SaCas9 AAVcc47-Ai9-sgRNA2-CB-SaCas9 AAVcc47-Ai9-sgRNA1-CB-SaCas9 AAV9-Ai9-sgRNA1-CB-SaCas9 --> AAV name --> Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to Asokan Aravind Last Updated Date: 2021-09-16 A dual vector strategy was employed: one delivering a single guide RNA and CB driven SaCas9, and another delivering the second guide RNA and CB driven SaCas9. This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=5) by intravenous injection in Ai9 mice. A total dose of 2e12vg was injected into each mouse (1e12vg each vector mixed 1:1) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
TadA-8e V106W Genome Editor - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-Pcsk9 --> compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV Catalytically impaired Cas fused to evolved TadA deaminase (TadA-8e V106W) Show Experiments (2) Pcsk9 adenine base editor efficiency in liver and nonliver tissue On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver
Cre Recombinase dose escalation study in Ai9 mice Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV --> AAV9-CMV-Cre AAVcc47-CMV-Cre --> AAV Asokan Aravind Last Updated Date: 2021-09-16 A single stranded cmv cre cassette was packaged into AAV9 or AAVcc47 and injected intravenously in Ai9 mice. We injected n=3 at three different doses (1e10, 1e11, 1e12 vg) and harvested organs 4 weeks post injection. Fluorescence intensity in liver, heart, and skeletal muscle was quantified with tiff based images in Image J and neuronal transduction from each vector was quantified at the 1e12vg dose by counting the number of tdTomato+ neurons and number of NeuN+ cells from multiple sections and images. Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
demo VV01-Cre Vector [Mouse] Matched on: --> AAV DEMO viral vector for demo purpose
demo VV05-Cre Vector [Mouse] Matched on: --> AAV DEMO viral vector for demo purpose
BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2 Vector - [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] Matched on: --> Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP --> AAV --> AAV BI28 (novel engineered variant) Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds Show Experiments (2) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Testing AAV5 for activation of tdTomato in mouse airway Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV.pU1a-SpCas9 H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV name --> Testing AAV5 for activation of tdTomato in mouse airway Gao Guang-Ping Last Updated Date: 2020-10-20 AAV2/5 mediated gene editing in the mouse airway was tested by deliverying SpCas9 and guide RNAs targeting the Ai9 transgene in Ai9 transgenic mice. Viral delivery was detected by GFP expression and gene editing quantified by tdTomato activation Show Project Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
demo VV04-Cre Vector [Mouse] Matched on: --> AAV DEMO viral vector for demo purpose
Ai9 mouse Model System - [In Vivo] [Small Animal Testing Center (SATC), AAV tropism, Delivery Systems Initiative] [Mouse] Matched on: --> SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV tropism --> AAV --> AAVrh8-ZsGreen-Cre AAVcc47-CMV-SaCas9 AAV9-CMV-SaCas9 AAV6-ZsGreen-Cre AAV9-CMV-Cre --> AAV --> AAV BI28 (novel engineered variant) --> AAV Tropism project Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Show Experiments (10) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Testing AAV5 for activation of tdTomato in mouse airway Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) AAV Tropism project Cre Recombinase dose escalation study in Ai9 mice Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV9-Ai9-sgRNA1 + sgRNA2 AAVcc47-Ai9-sgRNA1 + sgRNA2 AAVcc47-CMV-SaCas9 AAV9-CMV-SaCas9 --> single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV --> AAV name --> Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to Asokan Aravind Last Updated Date: 2021-09-16 A dual vector strategy was employed: one delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV cassettes). This strategy was evaluted with both AAV9 (n=3) and AAVcc47 (n=3) by intravenous injection in Ai9 mice. A total dose of 3e12vg was injected into each mouse (1.5e12vg each vector mixed 1:1) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> 3e11 vg/mouse of AAV-BI28:GFAP-SaCas9-WPRE-pA and 3e11 vg/mouse of AAV-BI28:GFAP-NLS-GFP-U6-L1-U6-R2 --> AAV --> AAV BI28 (novel engineered variant) Deverman Benjamin E Last Updated Date: 2021-04-17 3e11 vg/mouse of AAV-BI28:GFAP-SaCas9-WPRE-pA and 3e11 vg/mouse of AAV-BI28:GFAP-NLS-GFP-U6-L1-U6-R2 were codelivered intravenously to adult male and female Ai9 mice. Editing was assessed in brain sections 4 weeks later. Show Project Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System
Testing AAV5 for activation of tdTomato in HEK293T cells Experiment - [In Vitro] [Delivery Systems Initiative] [Human] Matched on: --> PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV shuttle plasmids expressing SpCas9 and guide RNAs targeting the Ai9 transgene were tested in HEK293T name --> Testing AAV5 for activation of tdTomato in HEK293T cells Gao Guang-Ping Last Updated Date: 2020-10-20 AAV shuttle plasmids expressing SpCas9 and guide RNAs targeting the Ai9 transgene were tested in HEK293T cells by transient transfection. Both delivery and gene editing were detected by fluorescence. Show Project Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector. Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV9_pTR_self comp 2xU6-Ai9 guides AAV9-CMV-SaCas9 AAVcc47_pTR_self comp 2xU6-Ai9 guides --> AAV name --> Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to Asokan Aravind Last Updated Date: 2021-09-16 A dual vector strategy was employed: one self complementary vector delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (single stranded vector). This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=4) by intravenous injection in Ai9 mice. A total dose of 4e12vg was injected into each mouse and vectors mixed in a 1:1 ratio of cas9 to guide RNA (2e12vg of CMV Sacas9 vector and 2e12vg of the self complementary sgRNA vector) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
C57BL/6 mouse (Asokan study) Model System - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV9-mCherry AAVcc47-mCherry AAVcc84-GFP AAVcc81-GFP AAV9-GFP --> AAV Show Experiments (2) Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Ai9 SaCas9 Guide A Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAVcc47-SaCas9-Ai9 --> AAV --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular This gRNA targets the Ai9 and related transgenes Show Experiments (1) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Ai9 SaCas9 Guide B Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAVcc47-SaCas9-Ai9 --> AAV --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular This gRNA targets the Ai9 and related transgenes Show Experiments (1) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
SauCas9 Genome Editor - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAVcc47-SaCas9-Ai9 --> AAV --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Show Experiments (1) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System Project [Delivery Systems Initiative] Matched on: name --> Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Deverman Benjamin E Last Updated Date: 2021-04-17 This project aims to advance the NIH Somatic Cell Genome Editing Program’s objective to identify novel delivery technologies that enable genome editing in therapeutically relevant somatic cell populations. We will use proven virus engineering methods to develop new vehicles that can deliver genome editing machinery throughout the adult mammalian central nervous system. Accomplishing this objective would pave the road for applying gene editing, and gene therapy more broadly, to the study and treatment of neurological and psychiatric disorders. Show Experiments (2) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
L1-unmodified Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb This gRNA targets the Ai9 and related transgenes Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAVcc84-GFP AAVcc81-GFP AAV9-GFP --> AAV Asokan Aravind Last Updated Date: 2020-11-19 C57BL/6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding a GFP reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification. Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
SaCas9 Genome Editor - [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 --> AAV --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb --> AAV BI28 (novel engineered variant) Show Experiments (3) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Deverman_Area Based Quantification of Editing Efficiency Protocol Protocol - [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 --> AAV --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb --> AAV BI28 (novel engineered variant) Procedure for non-IHC based image quantification of editing. Show Experiments (2) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Deverman_Comprehensive Methods Protocol - [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 --> Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue --> AAV --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb --> AAV BI28 (novel engineered variant) Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue processing and IHC. Show Experiments (2) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
L3-unmodified Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb This gRNA targets the Ai9 and related transgenes Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
SaLoxP2-unmodified Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2.gb This gRNA targets the mTmG, Ai9 and related transgenes at two sites Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Deverman_AAV Production and Administration Protocol Protocol - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 --> Published procedures for AAV production, delivery, and tissue imaging. Challis et al. Systemic AAV vectors for widespread and targeted gene delivery in rodents. --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb Published procedures for AAV production, delivery, and tissue imaging. Challis et al. Systemic AAV vectors for widespread and targeted gene delivery in rodents. Nat Protoc. 2019 Feb;14(2):379-414. doi: 10.1038/s41596-018-0097-3. Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
SaLoxP2-modified Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2.gb This gRNA targets the mTmG, Ai9 and related transgenes at two sites Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
BI28:AAV-GFAP-SaCas9-WPRE3-pA Vector - [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] Matched on: --> Novel engineered AAV BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein --> AAV --> AAV BI28 (novel engineered variant) Novel engineered AAV BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein (GFAP) promoter Show Experiments (2) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Ai9 mouse immortalized fibroblasts Model System - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb Immortalized fibroblasts made from Ai9 (B6.Cg-Gt(ROSA)26Sor^tm9(CAG-tdTomato)Hze/J) mice Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Chaikof-Associated Protocol 2_Off-target editing AND Primers for sequencing analysis Protocol - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-Pcsk9 --> compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV This protocol describes in vivo adminstration and subsequent analysis of off-target editing in the liver. Show Experiments (1) On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver
R1-unmodified Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb This gRNA targets the Ai9 and related transgenes Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
R2-unmodified Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb This gRNA targets the Ai9 and related transgenes Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Ai9 mouse (BCM) Model System - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAVcc47-Cre AAVcc47-SaCas9-Ai9 --> AAV --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Show Experiments (2) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
R1-modified Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb This gRNA targets the Ai9 and related transgenes Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
sgAi9R Guide - [In Vivo, In Vitro] [Delivery Systems Initiative] [Human, Mouse] Matched on: --> AAV.pU1a-SpCas9 PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV This sgRNA targets the Ai9 and related transgenes Show Experiments (3) Testing AAV5 for activation of tdTomato in HEK293T cells Testing AAV5 for activation of tdTomato in mouse airway Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Antibody - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV.pU1a-SpCas9 H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV Other Id: Thermo Fisher Scientific, MA5-15257 Anti-RFP (Mouse) Monoclonal Antibody, dilution used 1:300 Show Experiments (1) Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
eVLP Delivery System - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV engineered virus like particles Show Experiments (2) Pcsk9 adenine base editor efficiency in liver and nonliver tissue On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver
L2-unmodified Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb This gRNA targets the Ai9 and related transgenes Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
L1-modified Guide - [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 --> AAV --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb --> AAV BI28 (novel engineered variant) This gRNA targets the Ai9 and related transgenes Show Experiments (3) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
L2-modified Guide - [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 --> AAV --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb --> AAV BI28 (novel engineered variant) This gRNA targets the Ai9 and related transgenes Show Experiments (2) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
SaLoxP1-unmodified Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1 --> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1.gb This gRNA targets the mTmG, Ai9 and related transgenes at two sites Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) Vector - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV.pU1a-SpCas9 H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV AAV2/5 expressing SpyCas9. AAV2/5 expressing two sgRNAs under U6 promoter and eGFP Show Experiments (2) Testing AAV5 for activation of tdTomato in mouse airway Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Antibody - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV.pU1a-SpCas9 H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV Other Id: Santa Cruz, SC-23950 Anti-alpha Tubulin (Mouse) Monoclonal Antibody, dilution used 1:200 Show Experiments (1) Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Antibody - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV.pU1a-SpCas9 H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV Other Id: Rockland, 600- 401-379 Anti-RFP (Rabbit) Polyclonal Antibody Show Experiments (1) Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Heaney_SATC Tissue Processing, Imaging and Analysis Protocol - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAVcc47-Cre AAVcc47-SaCas9-Ai9 --> AAV --> AAV BI28 (novel engineered variant) --> Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Procedure for tissue preparation, imaging and analysis. Show Experiments (4) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Independent validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
HEK-293T with Ai9 transient reporter assay Model System - [In Vitro] [Delivery Systems Initiative] [Human] Matched on: --> PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) HEK-293T cells transfected with an Ai9 inducible transgene reporter plasmid used to test gene editing activity by fluorescence. HEK293T is an epithelial-like cell that was isolated from the kidney of a patient. Show Experiments (1) Testing AAV5 for activation of tdTomato in HEK293T cells
Antibody - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV.pU1a-SpCas9 H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV Other Id: Santa Cruz, SC-25555 Anti-CC10 (Rabbit) Polyclonal Antibody, dilution used 1:2,000 Show Experiments (1) Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Experiment - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb Deverman Benjamin E Last Updated Date: 2021-04-17 Reporter transgene activation by SaCas9 gRNA target and modified scaffold sequences by transient transfection in immortalized Ai9 mouse fibroblasts Show Project Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System
Lipofectamine 3000 Delivery System - [In Vitro] [Genome Editors, Delivery Systems Initiative] [Human] Matched on: --> PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) Lipid nanoparticle Show Experiments (2) Testing AAV5 for activation of tdTomato in HEK293T cells Testing newly discovered Biggie Phage editors in human cells
C57BL/6J mouse Model System - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-Pcsk9 --> compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV C57BL/6J WT mouse Show Experiments (3) Pcsk9 adenine base editor efficiency in liver and nonliver tissue On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice
L3-modifed Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3.gb This gRNA targets the Ai9 and related transgenes Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
R2-modified Guide - [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 --> AAV --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb --> AAV BI28 (novel engineered variant) This gRNA targets the Ai9 and related transgenes Show Experiments (2) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
pH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) Vector - [In Vitro] [Delivery Systems Initiative] [Human] Matched on: --> PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) AAV2/5 expressing SpyCas9. AAV2/5 expressing two sgRNAs under U6 promoter and eGFP Show Experiments (1) Testing AAV5 for activation of tdTomato in HEK293T cells
sgAi9L Guide - [In Vivo, In Vitro] [Delivery Systems Initiative] [Human, Mouse] Matched on: --> AAV.pU1a-SpCas9 PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV This sgRNA targets the Ai9 and related transgenes Show Experiments (3) Testing AAV5 for activation of tdTomato in HEK293T cells Testing AAV5 for activation of tdTomato in mouse airway Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
pAAV.pU1a-SpCas9 Vector - [In Vitro] [Delivery Systems Initiative] [Human] Matched on: --> PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS Show Experiments (1) Testing AAV5 for activation of tdTomato in HEK293T cells
Antibody - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV.pU1a-SpCas9 H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV Other Id: Rockland Cat# 600-401-379 Anti-RFP (RABBIT) Antibody Show Experiments (1) Testing AAV5 for activation of tdTomato in mouse airway
ssAAV5-sgB.saCas9 Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Antibody - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV.pU1a-SpCas9 H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) --> AAV Other Id: Cell Signaling Technology Cat# 2956 GFP (D5.1) XP Rabbit mAb antibody Show Experiments (1) Testing AAV5 for activation of tdTomato in mouse airway
SaLoxP1-modified Guide - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched on: --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1 --> AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1.gb This gRNA targets the mTmG, Ai9 and related transgenes at two sites Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
[Validation] Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice Experiment - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Heaney Jason D Last Updated Date: 2021-03-30 AAV5 encoding CRISPR/Cas editing machinery were delivered to the lungs of reporter mice by intratracheal instillation. After 4 weeks incubation, the mice were dissected and the lungs imaged for the presence of tdTomato fluorescence, indicating successful editing. Editing calculated by dividing the number of tdTomato+ red cells by the number of nuclei in each airway Show Project BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Gao Validation (Gao, Guang-Ping )
BCM_ssAAV5-Sa_sgB Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV This gRNA targets the Ai9 and related transgenes Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
SaCas9 Genome Editor - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
scAAV5-Cre-GFP Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
BCM_ssAAV5-Sp_sgB Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV This sgRNA targets the Ai9 and related transgenes Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Ai9-SauSpyCas9 mouse Model System - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Ai9-SauSpyCas9 is a version of Ai9 that has a single guide RNA sites on both sides of the STOP cassette. This is a Cre reporter allele that has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Ai9 mice express robust tdTomato fluorescence following Cre-mediated recombination. Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
SpCas9 Genome Editor - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Show Experiments (2) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice Independent validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
BCM_ssAAV5-Sp_sgA Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV This sgRNA targets the Ai9 and related transgenes Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
ssAAV5-sgA+sgB-U1A.GFP Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
ssAAV5-spCas9 Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice

149 results for AAV

Category	Type	Name	Description	Last Updated Date	View Associated..
Delivery System	Viral vector	AAV	See vector details		Show Experiments (2) AAV Tropism project Cre Recombinase dose escalation study in Ai9 mice
Experiment	In Vivo	AAV Tropism project	Ten AAV serotypes delivering Cre recombinase were tested by intravenous delivery into Ai9 mice and chacterized for biodistribution across 20 tissues by quantitative PCR and imaging	2023-02-10	Show Project SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice
Protocol		Conklin_Fast-Seq AAV Protocol	Published procedure for AAV composition measurement using fast-seq. Maynard et al. Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings. Hum Gene Ther Methods . 2019 Dec;30(6):195-205. doi: 10.1089/hgtb.2019.110.
Protocol		Heaney-SATC_Gao-Validation_Intratracheal Delivery of AAV in Mice	Procedure for Intratracheal (IT) delivery of AAV in mouse lung.		Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Project		SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice		2023-02-10	Show Experiments (1) AAV Tropism project
Project		Evolving High Potency AAV Vectors for Neuromuscular Genome Editing	Recombinant adeno-associated viruses (AAV) have emerged as safe and effective vectors for clinical gene therapy applications including systemic treatment of neuromuscular diseases such as Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD), and Giant Axonal Neuropathy (GAN) amongst others. However, genome editing in neuromuscular tissue, in particular, is challenging. The current proposal is on a comprehensive and innovative approach to evolve high potency AAV variants for systemic neuromuscular genome editing.	2020-11-19	Show Experiments (7) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Cre Recombinase dose escalation study in Ai9 mice Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Experiment	In Vivo	Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.	Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues.	2021-03-30	Show Project BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Asokan Validation (Asokan, Aravind)
Experiment	In Vivo	On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver	On-target editing compared to off-target editing at 14 CIRCLE seq nominated sites in livers of an adenine base editor delivered by engineered virus-like particles (BE-eVLPs). Treated mice vs. untreated vs. AAV was assessed one week after systemic administration of BE-eVLPs or AAV-Pcsk9 to C57BL/6 mice. DNA sequencing reads containing A-T to G-C mutations within protospacer positions 4-10.	2022-04-15	Show Project Delivery Technologies for In Vivo Genome Editing
Guide		AAVS1_site_12	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_13	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_04	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_11	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_01	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_05	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_06	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_08	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_10	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_03	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_09	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_02	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_07	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_14	Targets AAVS1 safe harbor locus		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Vector	Viral Vector	AAV6-ZsGreen-Cre	AAV backbone with a bi-directional promoter driving zsGreen and Cre		Show Experiments (1) AAV Tropism project
Vector	Viral Vector	AAV9-Ai9-sgRNA2-CB-SaCas9	AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus		Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
Vector	Viral Vector	AAV9_pTR_self comp 2xU6-Ai9 guides	AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene		Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Vector	Viral Vector	AAV9-ZsGreen-Cre	AAV backbone with a bi-directional promoter driving zsGreen and Cre		Show Experiments (1) AAV Tropism project
Vector	Viral Vector	AAVcc47-CMV-SaCas9	AAVcc47 delivering CMV driven SaCas9		Show Experiments (2) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Vector	Viral Vector	AAVcc47_pTR_self comp 2xU6-Ai9 guides	AAVcc47 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene		Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Vector	Viral Vector	AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV7-ZsGreen-Cre	AAV backbone with a bi-directional promoter driving zsGreen and Cre		Show Experiments (1) AAV Tropism project
Vector	Viral Vector	AAV8-ZsGreen-Cre	AAV backbone with a bi-directional promoter driving zsGreen and Cre		Show Experiments (1) AAV Tropism project
Vector	Viral Vector	AAVcc47-Ai9-sgRNA1-CB-SaCas9	AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus		Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
Vector	Viral Vector	AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV-Pcsk9			Show Experiments (1) On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver
Vector	Viral Vector	AAV9-Ai9-sgRNA1-CB-SaCas9	AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus		Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
Vector	Viral Vector	AAV9-CMV-SaCas9	AAV serotype 9 delivering CMV driven SaCas9		Show Experiments (3) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Vector	Viral Vector	AAVcc47-Cre	AAV2/5 expressing Cre recombinase		Show Experiments (1) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Vector	Viral Vector	AAVcc47-SaCas9-Ai9	AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter		Show Experiments (1) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Vector	Viral Vector	AAVcc84-GFP	AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter		Show Experiments (1) Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Vector	Viral Vector	AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV5-ZsGreen-Cre	AAV backbone with a bi-directional promoter driving zsGreen and Cre		Show Experiments (1) AAV Tropism project
Vector	Viral Vector	AAV9-Ai9-sgRNA1 + sgRNA2	AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus		Show Experiments (2) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Vector	Viral Vector	AAVcc47-Ai9-sgRNA2-CB-SaCas9	AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus		Show Experiments (1) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
Vector	Viral Vector	AAVcc47-CMV-Cre	AAVcc47 delivering CMV Cre Recombinase		Show Experiments (1) Cre Recombinase dose escalation study in Ai9 mice
Vector	Viral Vector	AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAVrh10-ZsGreen-Cre	AAV backbone with a bi-directional promoter driving zsGreen and Cre		Show Experiments (1) AAV Tropism project
Vector	Viral Vector	AAVrh74-ZsGreen-Cre	AAV backbone with a bi-directional promoter driving zsGreen and Cre		Show Experiments (1) AAV Tropism project
Vector	Viral Vector	AAVrh8-ZsGreen-Cre	AAV backbone with a bi-directional promoter driving zsGreen and Cre		Show Experiments (1) AAV Tropism project
Vector	Viral Vector	AAV4-ZsGreen-Cre	AAV backbone with a bi-directional promoter driving zsGreen and Cre		Show Experiments (1) AAV Tropism project
Vector	Viral Vector	AAV9-GFP	AAV2/9 self complementary vector expressing GFP driven by CBh promoter		Show Experiments (1) Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Vector	Viral Vector	AAV9-mCherry	AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter		Show Experiments (1) Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Vector	Viral Vector	AAVcc81-GFP	AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter		Show Experiments (1) Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Vector	Viral Vector	AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAVcc47-Ai9-sgRNA1 + sgRNA2	AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus		Show Experiments (2) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Vector	Viral Vector	AAV9-CMV-Cre	AAV serotype 9 delivering CMV Cre Recombinase		Show Experiments (1) Cre Recombinase dose escalation study in Ai9 mice
Vector	Viral Vector	AAVcc47-mCherry	AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter		Show Experiments (1) Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Vector	Viral Vector	AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV.pU1a-SpCas9	Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS		Show Experiments (2) Testing AAV5 for activation of tdTomato in mouse airway Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Vector	Viral Vector	AAV3b-ZsGreen-Cre	AAV backbone with a bi-directional promoter driving zsGreen and Cre		Show Experiments (1) AAV Tropism project
Vector	Viral Vector	AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Protocol		Tsai_T Cell Transfection Protocol	Procedure for T cell transfection.		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Vector	Viral Vector	demo VV03-Cre	DEMO viral vector for demo purpose
Vector	Viral Vector	demo VV02-Cre	DEMO viral vector for demo purpose
Experiment	In Vivo	Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)	A dual vector strategy was employed: one delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV cassettes). This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=5) by intravenous injection in Ai9 mice. A total dose of 4e12vg was injected into each mouse and vectors mixed in a 1:4 ratio of cas9 to guide RNA (1e12vg of CMV Sacas9 vector and 3e12vg of the sgRNA vector) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart.	2021-09-16	Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Experiment	In Vivo	Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain	Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing. Editing detected and quantified in brain by generation of tdTomato fluorescent protein signal from Ai9 reporter mice	2023-05-10	Show Project BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin)
Experiment	In Vivo	Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells	AAV2/5 mediated gene editing in the mouse airway was tested by deliverying SpCas9 and guide RNAs targeting the Ai9 transgene in Ai9 transgenic mice. Gene editing quantified by tdTomato activation and cell specific markers for club and ciliated cell types.	2021-09-21	Show Project Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
Genome Editor	Cas Nuclease	SaCas9-Lagor			Show Experiments (4) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Experiment	In Vivo	Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.	C57BL/6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding an mCherry reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification.	2020-11-19	Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Genome Editor	Recombinase	Cre recombinase	Cre recombinase delivered by AAV (see vector details)		Show Experiments (3) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Experiment	In Vitro	A novel human T cell platform to define biological adverse effects of genome editing	110 guide RNAs and SpCas9 were transfected into human T-cells. Indel rates were measured by targeted amplicon deep sequencing.	2020-12-09	Show Project A Novel Human T-Cell Platform to Define Biological Effects of Genome Editing
Delivery System	Commercial Reagent	P3 Nucleofection Kit	Nuceleofection kit to be used with Lonza's nucleofection system.		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Model System	Cell	CD4/CD8 Human Primary T cell	CD4/CD8 Human Primary T cell		Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Genome Editor	Cas Nuclease	SpCas9	HA-SV40NLS-SpCas9-SV40NLS		Show Experiments (4) Testing AAV5 for activation of tdTomato in HEK293T cells Testing AAV5 for activation of tdTomato in mouse airway A novel human T cell platform to define biological adverse effects of genome editing Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Experiment	In Vivo	Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)	A dual vector strategy was employed: one delivering a single guide RNA and CB driven SaCas9, and another delivering the second guide RNA and CB driven SaCas9. This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=5) by intravenous injection in Ai9 mice. A total dose of 2e12vg was injected into each mouse (1e12vg each vector mixed 1:1) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart.	2021-09-16	Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Genome Editor	Base Editor (SpCas9)	TadA-8e V106W	Catalytically impaired Cas fused to evolved TadA deaminase (TadA-8e V106W)		Show Experiments (2) Pcsk9 adenine base editor efficiency in liver and nonliver tissue On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver
Experiment	In Vivo	Cre Recombinase dose escalation study in Ai9 mice	A single stranded cmv cre cassette was packaged into AAV9 or AAVcc47 and injected intravenously in Ai9 mice. We injected n=3 at three different doses (1e10, 1e11, 1e12 vg) and harvested organs 4 weeks post injection. Fluorescence intensity in liver, heart, and skeletal muscle was quantified with tiff based images in Image J and neuronal transduction from each vector was quantified at the 1e12vg dose by counting the number of tdTomato+ neurons and number of NeuN+ cells from multiple sections and images.	2021-09-16	Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Vector	Viral Vector	demo VV01-Cre	DEMO viral vector for demo purpose
Vector	Viral Vector	demo VV05-Cre	DEMO viral vector for demo purpose
Vector	Viral Vector	BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2	Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds		Show Experiments (2) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Experiment	In Vivo	Testing AAV5 for activation of tdTomato in mouse airway	AAV2/5 mediated gene editing in the mouse airway was tested by deliverying SpCas9 and guide RNAs targeting the Ai9 transgene in Ai9 transgenic mice. Viral delivery was detected by GFP expression and gene editing quantified by tdTomato activation	2020-10-20	Show Project Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
Vector	Viral Vector	demo VV04-Cre	DEMO viral vector for demo purpose
Model System	Animal	Ai9 mouse	Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.		Show Experiments (10) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Testing AAV5 for activation of tdTomato in mouse airway Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) AAV Tropism project Cre Recombinase dose escalation study in Ai9 mice Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Experiment	In Vivo	Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter)	A dual vector strategy was employed: one delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV cassettes). This strategy was evaluted with both AAV9 (n=3) and AAVcc47 (n=3) by intravenous injection in Ai9 mice. A total dose of 3e12vg was injected into each mouse (1.5e12vg each vector mixed 1:1) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart.	2021-09-16	Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Experiment	In Vivo	Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.	3e11 vg/mouse of AAV-BI28:GFAP-SaCas9-WPRE-pA and 3e11 vg/mouse of AAV-BI28:GFAP-NLS-GFP-U6-L1-U6-R2 were codelivered intravenously to adult male and female Ai9 mice. Editing was assessed in brain sections 4 weeks later.	2021-04-17	Show Project Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System
Experiment	In Vitro	Testing AAV5 for activation of tdTomato in HEK293T cells	AAV shuttle plasmids expressing SpCas9 and guide RNAs targeting the Ai9 transgene were tested in HEK293T cells by transient transfection. Both delivery and gene editing were detected by fluorescence.	2020-10-20	Show Project Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
Experiment	In Vivo	Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.	A dual vector strategy was employed: one self complementary vector delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (single stranded vector). This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=4) by intravenous injection in Ai9 mice. A total dose of 4e12vg was injected into each mouse and vectors mixed in a 1:1 ratio of cas9 to guide RNA (2e12vg of CMV Sacas9 vector and 2e12vg of the self complementary sgRNA vector) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart.	2021-09-16	Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Model System	Animal	C57BL/6 mouse (Asokan study)			Show Experiments (2) Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Guide		Ai9 SaCas9 Guide A	This gRNA targets the Ai9 and related transgenes		Show Experiments (1) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Guide		Ai9 SaCas9 Guide B	This gRNA targets the Ai9 and related transgenes		Show Experiments (1) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Genome Editor	Cas Nuclease	SauCas9			Show Experiments (1) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Project		Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System	This project aims to advance the NIH Somatic Cell Genome Editing Program’s objective to identify novel delivery technologies that enable genome editing in therapeutically relevant somatic cell populations. We will use proven virus engineering methods to develop new vehicles that can deliver genome editing machinery throughout the adult mammalian central nervous system. Accomplishing this objective would pave the road for applying gene editing, and gene therapy more broadly, to the study and treatment of neurological and psychiatric disorders.	2021-04-17	Show Experiments (2) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Guide		L1-unmodified	This gRNA targets the Ai9 and related transgenes		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Experiment	In Vivo	Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.	C57BL/6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding a GFP reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification.	2020-11-19	Show Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Genome Editor	Cas Nuclease

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149 results for AAV

149 results for AAV

[Validation] Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.

[Validation] Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain

[Validation] Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice