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AAV - Delivery System - [In Vivo] [DCC, Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing name --> AAV --> AAV --> AAV --> AAV9-CMV-Cre --> AAV name --> AAV See vector details Experiments Cre Recombinase dose escalation study in Ai9 mice MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
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. Experiments 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] 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: --> Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAVcc47-Cre AAVcc47-SaCas9-Ai9 --> Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. --> AAV name --> Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing 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. Projects 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. Projects Delivery Technologies for In Vivo 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 Experiments 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 Experiments 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 Experiments 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 Experiments 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 Experiments 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 Experiments 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 Experiments 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 Experiments 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 Experiments 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 Experiments 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 Experiments 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 Experiments A novel human T cell platform to define biological adverse effects of 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 Experiments 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 Experiments A novel human T cell platform to define biological adverse effects of genome editing
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 Experiments 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 Experiments Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
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 Experiments 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 Experiments Testing AAV5 for activation of tdTomato in mouse airway Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
AAVcc47-Cre - Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: symbol --> AAVcc47-Cre --> AAVcc47-Cre --> AAV --> Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing AAV2/5 expressing Cre recombinase Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
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 Experiments 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-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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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 Experiments 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-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 Experiments 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 Experiments 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-SaCas9-Ai9 - Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: symbol --> AAVcc47-SaCas9-Ai9 --> AAVcc47-SaCas9-Ai9 --> AAV --> Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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 Experiments 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-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 Experiments 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-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 Experiments 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-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 Experiments 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 Experiments 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 Experiments 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-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 Experiments 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-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 Experiments Cre Recombinase dose escalation study in Ai9 mice
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 Experiments 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)
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 Experiments Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
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 Experiments 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 Experiments 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 Experiments 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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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 Experiments 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 Experiments Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
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 Experiments Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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 Experiments 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-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 Experiments Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
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 Experiments 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-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 Experiments 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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Ai9 SaCas9 Guide A - Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAVcc47-SaCas9-Ai9 --> AAV --> Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing This gRNA targets the Ai9 and related transgenes Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
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. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only - Experiment - [In Vivo] [DCC] [Mouse] Matched on: --> AAV Geurts Aron Last Updated Date: 2023-02-21 MOCK STUDY for demonstration purpose only. Data is fake. Five viral vector serotypes delivering Cre recombinase were tested by intravenous delivery into Ai9 mice and chacterized for biodistribution across 20 tissues by quantitative PCR and imaging. Cirulating IL-6 levels as well as cellular toxicity in the kidney were assessed 1-week post inoculation. Projects DEMO STUDY For Toolkit demonstration purposes only
demo VV03-Cre - Vector - [In Vivo] [DCC] [Mouse] Matched on: --> AAV DEMO viral vector for demo purpose Experiments MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
demo VV05-Cre - Vector - [In Vivo] [DCC] [Mouse] Matched on: --> AAV DEMO viral vector for demo purpose Experiments MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
Ai9 SaCas9 Guide B - Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAVcc47-SaCas9-Ai9 --> AAV --> Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing This gRNA targets the Ai9 and related transgenes Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
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. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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. Projects Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
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 Projects Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
Deverman_AAV_production_and_administration_Nat Protocols-Challis_et_al_2019.pdf - Protocol Matched on: --> Published procedures for AAV production, delivery, and tissue imaging. Challis et al. Systemic AAV vectors for widespread and targeted gene delivery in rodents. 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.
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. Projects A Novel Human T-Cell Platform to Define Biological 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 Experiments 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
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. Experiments 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 Experiments A novel human T cell platform to define biological adverse effects of 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 --> Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Cre recombinase delivered by AAV (see vector details) Experiments 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 Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
demo VV01-Cre - Vector - [In Vivo] [DCC] [Mouse] Matched on: --> AAV DEMO viral vector for demo purpose Experiments MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
demo VV02-Cre - Vector - [In Vivo] [DCC] [Mouse] Matched on: --> AAV DEMO viral vector for demo purpose Experiments MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
demo VV04-Cre - Vector - [In Vivo] [DCC] [Mouse] Matched on: --> AAV DEMO viral vector for demo purpose Experiments MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
Heaney-SATC_Gao-Validation_Intratracheal_Delivery_of_AAV_in_Mice.pdf - Protocol Matched on: --> Procedure for Intratracheal (IT) delivery of AAV in mouse lung. Procedure for Intratracheal (IT) delivery of AAV in mouse lung.
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. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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. Projects Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
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 C57/BL6 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. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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 C57/BL6 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. Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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 Experiments 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.
Ai9 mouse - Model System - [In Vivo] [Small Animal Testing Center (SATC), DCC, Delivery Systems Initiative] [Mouse] Matched on: --> Evolving High Potency AAV Vectors for Neuromuscular Genome Editing --> AAV --> AAV9-CMV-Cre AAVcc47-CMV-SaCas9 AAV9-CMV-SaCas9 AAV.pU1a-SpCas9 AAVcc47-CMV-Cre --> AAV --> AAV BI28 (novel engineered variant) 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. Experiments 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) Cre Recombinase dose escalation study in Ai9 mice MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only Second site 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.
[Validation] Second site 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 --> Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to --> AAV BI28 (novel engineered variant) Heaney Jason D Last Updated Date: 2021-08-08 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 Projects BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin)
SauCas9 - Genome Editor - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAVcc47-SaCas9-Ai9 --> AAV --> Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Experiments Validation for Asokan Delivery Team: 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 Experiments 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.
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. Experiments 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.pdf - Protocol Matched on: --> Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue processing and IHC.
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 Experiments 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 Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
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) Experiments 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 Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
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 Experiments 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 Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb This gRNA targets the Ai9 and related transgenes Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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. Projects Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System
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 Experiments 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 Experiments 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 --> Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing 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. Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
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 Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
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-gRNA-LoxP-Sa-L3.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb --> AAV BI28 (novel engineered variant) This gRNA targets the Ai9 and related transgenes Experiments 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
- 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 Experiments Testing AAV5 for activation of tdTomato in mouse airway
- 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 Experiments Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
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) Experiments 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
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 Experiments 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
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 Experiments 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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
- 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 Experiments Testing AAV5 for activation of tdTomato in mouse airway
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 Experiments 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
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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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 Experiments 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
- 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 Experiments Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
SaCas9 - Genome Editor - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
ssAAV5-sgB.saCas9 - Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Experiments 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 Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
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 Projects Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System
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 Experiments 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-L1.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb This gRNA targets the Ai9 and related transgenes Experiments 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 Experiments Testing AAV5 for activation of tdTomato in HEK293T cells
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 Experiments 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 Experiments 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 Experiments Testing AAV5 for activation of tdTomato in HEK293T cells
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 Experiments Testing AAV5 for activation of tdTomato in mouse airway Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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 Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
- 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 Experiments 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 Experiments Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Conklin_Fast-Seq_AAV.pdf - Protocol Matched on: --> Published procedure for AAV composition measurement using fast-seq. Maynard et al. 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.
BCM_ssAAV5-Sp_sgA - Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV This sgRNA targets the Ai9 and related transgenes Experiments 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. Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
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 Experiments Testing AAV5 for activation of tdTomato in HEK293T cells Testing newly discovered Biggie Phage editors in human cells
[Validation] 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 Projects BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Gao Validation (Gao, Guang-Ping )
scAAV5-Cre-GFP - Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV Experiments 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 Experiments 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 Experiments 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 Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice Validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
BCM_ssAAV5-Sa_sgB - Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched on: --> AAV This gRNA targets the Ai9 and related transgenes Experiments Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice

134 results for AAV

Category	Type	Name	Description	Last Updated Date	View Associated..
Delivery System	Viral vector	AAV	See vector details		Experiments Cre Recombinase dose escalation study in Ai9 mice MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
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	Experiments 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	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	Projects 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	Projects Delivery Technologies for In Vivo Genome Editing
Guide		AAVS1_site_01	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_02	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_05	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_08	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_10	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_13	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_14	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_04	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_09	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_11	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_06	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_03	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_12	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Guide		AAVS1_site_07	Targets AAVS1 safe harbor locus		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Vector	Viral Vector	AAV9-GFP	AAV2/9 self complementary vector expressing GFP driven by CBh promoter		Experiments 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		Experiments 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-R2	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Experiments 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		Experiments 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	AAVcc47-Cre	AAV2/5 expressing Cre recombinase		Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Vector	Viral Vector	AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Experiments 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-L3	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV9-Ai9-sgRNA1 + sgRNA2	AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus		Experiments 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-CMV-SaCas9	AAVcc47 delivering CMV driven SaCas9		Experiments 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		Experiments 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-SaCas9-Ai9	AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter		Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Vector	Viral Vector	AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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		Experiments 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-Ai9-sgRNA2-CB-SaCas9	AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus		Experiments 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-L1	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Experiments 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-L2	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Experiments 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		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV-Pcsk9			Experiments 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-sgRNA2-CB-SaCas9	AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus		Experiments 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-Cre	AAV serotype 9 delivering CMV Cre Recombinase		Experiments Cre Recombinase dose escalation study in Ai9 mice
Vector	Viral Vector	AAVcc47-Ai9-sgRNA1 + sgRNA2	AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus		Experiments 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	AAVcc81-GFP	AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter		Experiments 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-L3	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Experiments 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		Experiments 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		Experiments 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		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAVcc47-CMV-Cre	AAVcc47 delivering CMV Cre Recombinase		Experiments 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		Experiments Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Vector	Viral Vector	AAVcc84-GFP	AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter		Experiments Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
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		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	AAV9-CMV-SaCas9	AAV serotype 9 delivering CMV driven SaCas9		Experiments 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-Ai9-sgRNA1-CB-SaCas9	AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus		Experiments 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-Ai9-sgRNA1-CB-SaCas9	AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus		Experiments 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-LoxP2	AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA		Experiments 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		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Guide		Ai9 SaCas9 Guide A	This gRNA targets the Ai9 and related transgenes		Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
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	Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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	Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Experiment	In Vivo	MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only	MOCK STUDY for demonstration purpose only. Data is fake. Five viral vector serotypes delivering Cre recombinase were tested by intravenous delivery into Ai9 mice and chacterized for biodistribution across 20 tissues by quantitative PCR and imaging. Cirulating IL-6 levels as well as cellular toxicity in the kidney were assessed 1-week post inoculation.	2023-02-21	Projects DEMO STUDY For Toolkit demonstration purposes only
Vector	Viral Vector	demo VV03-Cre	DEMO viral vector for demo purpose		Experiments MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
Vector	Viral Vector	demo VV05-Cre	DEMO viral vector for demo purpose		Experiments MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
Guide		Ai9 SaCas9 Guide B	This gRNA targets the Ai9 and related transgenes		Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
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	Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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	Projects Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
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	Projects Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
Protocol		Deverman_AAV_production_and_administration_Nat Protocols-Challis_et_al_2019.pdf	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.
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	Projects A Novel Human T-Cell Platform to Define Biological Effects of Genome Editing
Genome Editor	Cas Nuclease	SpCas9	HA-SV40NLS-SpCas9-SV40NLS		Experiments 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
Delivery System	Commercial Reagent	P3 Nucleofection Kit	Nuceleofection kit to be used with Lonza's nucleofection system.		Experiments 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		Experiments A novel human T cell platform to define biological adverse effects of genome editing
Genome Editor	Recombinase	Cre recombinase	Cre recombinase delivered by AAV (see vector details)		Experiments 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 Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Vector	Viral Vector	demo VV01-Cre	DEMO viral vector for demo purpose		Experiments MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
Vector	Viral Vector	demo VV02-Cre	DEMO viral vector for demo purpose		Experiments MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
Vector	Viral Vector	demo VV04-Cre	DEMO viral vector for demo purpose		Experiments MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only
Protocol		Heaney-SATC_Gao-Validation_Intratracheal_Delivery_of_AAV_in_Mice.pdf	Procedure for Intratracheal (IT) delivery of AAV in mouse lung.
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	Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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	Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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	Projects Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases
Experiment	In Vivo	Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.	C57/BL6 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	Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Experiment	In Vivo	Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.	C57/BL6 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	Projects Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Genome Editor	Cas Nuclease	SaCas9-Lagor			Experiments 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.
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.		Experiments 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) Cre Recombinase dose escalation study in Ai9 mice MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only Second site 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	Second site 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	2021-08-08	Projects BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin)
Genome Editor	Cas Nuclease	SauCas9			Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.
Model System	Animal	C57BL/6 mouse (Asokan study)			Experiments 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.
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	Experiments 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
Protocol		Deverman_Comprehensive_methods.pdf	Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue processing and IHC.
Guide		L1-modified	This gRNA targets the Ai9 and related transgenes		Experiments 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 Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Genome Editor	Cas Nuclease	SaCas9			Experiments 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 Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Guide		L3-unmodified	This gRNA targets the Ai9 and related transgenes		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Vector	Viral Vector	BI28:AAV-GFAP-SaCas9-WPRE3-pA	Novel engineered AAV BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein (GFAP) promoter		Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Guide		R1-modified	This gRNA targets the Ai9 and related transgenes		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Guide		R1-unmodified	This gRNA targets the Ai9 and related transgenes		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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	Projects Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System
Guide		SaLoxP1-unmodified	This gRNA targets the mTmG, Ai9 and related transgenes at two sites		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Guide		SaLoxP2-modified	This gRNA targets the mTmG, Ai9 and related transgenes at two sites		Experiments Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Model System	Animal	Ai9 mouse (BCM)	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.		Experiments Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
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		Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Guide		R2-modified	This gRNA targets the Ai9 and related transgenes		Experiments 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

Contact SCGE

134 results for AAV

134 results for AAV

AAV - Delivery System - [In Vivo] [DCC, Delivery Systems Initiative] [Mouse]

Evolving High Potency AAV Vectors for Neuromuscular Genome Editing - Project [Delivery Systems Initiative]

[Validation] Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. - Experiment - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]

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]

AAVS1_site_01 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_02 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_05 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_08 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_10 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_13 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_14 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_04 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_09 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_11 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_06 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_03 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_12 - Guide - [In Vitro] [Biological Systems] [Human]

AAVS1_site_07 - Guide - [In Vitro] [Biological Systems] [Human]

AAV9-GFP - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAV9-mCherry - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV.pU1a-SpCas9 - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAVcc47-Cre - Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV9-Ai9-sgRNA1 + sgRNA2 - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAVcc47-CMV-SaCas9 - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAVcc47_pTR_self comp 2xU6-Ai9 guides - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAVcc47-SaCas9-Ai9 - Vector - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV9_pTR_self comp 2xU6-Ai9 guides - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAVcc47-Ai9-sgRNA2-CB-SaCas9 - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV-Pcsk9 - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAV9-Ai9-sgRNA2-CB-SaCas9 - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAV9-CMV-Cre - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAVcc47-Ai9-sgRNA1 + sgRNA2 - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAVcc81-GFP - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAVcc47-CMV-Cre - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAVcc47-mCherry - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAVcc84-GFP - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV9-CMV-SaCas9 - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAVcc47-Ai9-sgRNA1-CB-SaCas9 - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAV9-Ai9-sgRNA1-CB-SaCas9 - Vector - [In Vivo] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1 - Vector - [In Vitro] [Delivery Systems Initiative] [Mouse]

Ai9 SaCas9 Guide A - Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]

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]

Cre Recombinase dose escalation study in Ai9 mice - Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse]

*MOCK VIRUS TROPISM STUDY* For Toolkit demonstration purposes only - Experiment - [In Vivo] [DCC] [Mouse]

demo VV03-Cre - Vector - [In Vivo] [DCC] [Mouse]

demo VV05-Cre - Vector - [In Vivo] [DCC] [Mouse]

Ai9 SaCas9 Guide B - Guide - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]

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]

Testing AAV5 for activation of tdTomato in HEK293T cells - Experiment - [In Vitro] [Delivery Systems Initiative] [Human]

Testing AAV5 for activation of tdTomato in mouse airway - Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse]

Deverman_AAV_production_and_administration_Nat Protocols-Challis_et_al_2019.pdf - Protocol

A novel human T cell platform to define biological adverse effects of genome editing - Experiment - [In Vitro] [Biological Systems] [Human]

SpCas9 - Genome Editor - [In Vivo, In Vitro] [Delivery Systems Initiative, Biological Systems] [Human, Mouse]

P3 Nucleofection Kit - Delivery System - [In Vitro] [Biological Systems] [Human]

CD4/CD8 Human Primary T cell - Model System - [In Vitro] [Biological Systems] [Human]

Cre recombinase - Genome Editor - [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse]

demo VV01-Cre - Vector - [In Vivo] [DCC] [Mouse]

demo VV02-Cre - Vector - [In Vivo] [DCC] [Mouse]

demo VV04-Cre - Vector - [In Vivo] [DCC] [Mouse]

Heaney-SATC_Gao-Validation_Intratracheal_Delivery_of_AAV_in_Mice.pdf - Protocol

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]

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]

Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells - Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse]

Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. - Experiment - [In Vivo] [Delivery Systems Initiative] [Mouse]

MOCK VIRUS TROPISM STUDY For Toolkit demonstration purposes only - Experiment - [In Vivo] [DCC] [Mouse]