134 results for AAV

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
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 NIH Report
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.
[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.
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.
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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.
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.
*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.
demo VV03-Cre   - Vector  - [In Vivo] [DCC] [Mouse]
Matched on:  -->  AAV
DEMO viral vector for demo purpose
demo VV05-Cre   - Vector  - [In Vivo] [DCC] [Mouse]
Matched on:  -->  AAV
DEMO viral vector for demo purpose
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
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.
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.
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
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.
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
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.
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
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)
demo VV01-Cre   - Vector  - [In Vivo] [DCC] [Mouse]
Matched on:  -->  AAV
DEMO viral vector for demo purpose
demo VV02-Cre   - Vector  - [In Vivo] [DCC] [Mouse]
Matched on:  -->  AAV
DEMO viral vector for demo purpose
demo VV04-Cre   - Vector  - [In Vivo] [DCC] [Mouse]
Matched on:  -->  AAV
DEMO viral vector for demo purpose
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.
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.
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.
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.
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.
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
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.
[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
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
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
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 NIH Report
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.
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
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)
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
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
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
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
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.
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
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
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.
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
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
  - 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
  - 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
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)
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
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
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
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
  - 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
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
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
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
  - 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
SaCas9   - Genome Editor  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV
ssAAV5-sgB.saCas9   - Vector  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV
ssAAV5-spCas9   - Vector  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV
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
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
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
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
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
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
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
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
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
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
  - 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
  - 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
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
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.
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
[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
scAAV5-Cre-GFP   - Vector  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV
ssAAV5-sgA+sgB-U1A.GFP   - Vector  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV
BCM_ssAAV5-Sp_sgB   - Guide  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV
This sgRNA targets the Ai9 and related transgenes
BCM_ssAAV5-Sa_sgB   - Guide  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV
This gRNA targets the Ai9 and related transgenes

134 results for AAV

Category Type Name Description Last Updated Date View Associated..
Delivery System Viral vector AAV See vector details
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
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
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
Guide AAVS1_site_01 Targets AAVS1 safe harbor locus
Guide AAVS1_site_02 Targets AAVS1 safe harbor locus
Guide AAVS1_site_05 Targets AAVS1 safe harbor locus
Guide AAVS1_site_08 Targets AAVS1 safe harbor locus
Guide AAVS1_site_10 Targets AAVS1 safe harbor locus
Guide AAVS1_site_13 Targets AAVS1 safe harbor locus
Guide AAVS1_site_14 Targets AAVS1 safe harbor locus
Guide AAVS1_site_04 Targets AAVS1 safe harbor locus
Guide AAVS1_site_09 Targets AAVS1 safe harbor locus
Guide AAVS1_site_11 Targets AAVS1 safe harbor locus
Guide AAVS1_site_06 Targets AAVS1 safe harbor locus
Guide AAVS1_site_03 Targets AAVS1 safe harbor locus
Guide AAVS1_site_12 Targets AAVS1 safe harbor locus
Guide AAVS1_site_07 Targets AAVS1 safe harbor locus
Vector Viral Vector AAV9-GFP AAV2/9 self complementary vector expressing GFP driven by CBh promoter
Vector Viral Vector AAV9-mCherry AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter
Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
Vector Viral Vector AAV.pU1a-SpCas9 Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS
Vector Viral Vector AAVcc47-Cre AAV2/5 expressing Cre recombinase
Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
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
Vector Viral Vector AAV9-Ai9-sgRNA1 + sgRNA2 AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus
Vector Viral Vector AAVcc47-CMV-SaCas9 AAVcc47 delivering CMV driven SaCas9
Vector Viral Vector AAVcc47_pTR_self comp 2xU6-Ai9 guides AAVcc47 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene
Vector Viral Vector AAVcc47-SaCas9-Ai9 AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter
Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
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
Vector Viral Vector AAVcc47-Ai9-sgRNA2-CB-SaCas9 AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
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
Vector Viral Vector AAV-Pcsk9
Vector Viral Vector AAV9-Ai9-sgRNA2-CB-SaCas9 AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAV9-CMV-Cre AAV serotype 9 delivering CMV Cre Recombinase
Vector Viral Vector AAVcc47-Ai9-sgRNA1 + sgRNA2 AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus
Vector Viral Vector AAVcc81-GFP AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter
Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
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
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
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
Vector Viral Vector AAVcc47-CMV-Cre AAVcc47 delivering CMV Cre Recombinase
Vector Viral Vector AAVcc47-mCherry AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter
Vector Viral Vector AAVcc84-GFP AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter
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
Vector Viral Vector AAV9-CMV-SaCas9 AAV serotype 9 delivering CMV driven SaCas9
Vector Viral Vector AAVcc47-Ai9-sgRNA1-CB-SaCas9 AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAV9-Ai9-sgRNA1-CB-SaCas9 AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
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
Guide Ai9 SaCas9 Guide A This gRNA targets the Ai9 and related transgenes
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
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
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
Vector Viral Vector demo VV03-Cre DEMO viral vector for demo purpose
Vector Viral Vector demo VV05-Cre DEMO viral vector for demo purpose
Guide Ai9 SaCas9 Guide B This gRNA targets the Ai9 and related transgenes
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
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
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
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
Genome Editor Cas Nuclease SpCas9 HA-SV40NLS-SpCas9-SV40NLS
Delivery System Commercial Reagent P3 Nucleofection Kit Nuceleofection kit to be used with Lonza's nucleofection system.
Model System Cell CD4/CD8 Human Primary T cell CD4/CD8 Human Primary T cell
Genome Editor Recombinase Cre recombinase Cre recombinase delivered by AAV (see vector details)
Vector Viral Vector demo VV01-Cre DEMO viral vector for demo purpose
Vector Viral Vector demo VV02-Cre DEMO viral vector for demo purpose
Vector Viral Vector demo VV04-Cre DEMO viral vector for demo purpose
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
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
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
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
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
Genome Editor Cas Nuclease SaCas9-Lagor
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.
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
Genome Editor Cas Nuclease SauCas9
Model System Animal C57BL/6 mouse (Asokan study)
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
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
Genome Editor Cas Nuclease SaCas9
Guide L3-unmodified This gRNA targets the Ai9 and related transgenes
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
Guide R1-modified This gRNA targets the Ai9 and related transgenes
Guide R1-unmodified This gRNA targets the Ai9 and related transgenes
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
Guide SaLoxP1-unmodified This gRNA targets the mTmG, Ai9 and related transgenes at two sites
Guide SaLoxP2-modified This gRNA targets the mTmG, Ai9 and related transgenes at two sites
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.
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
Guide R2-modified This gRNA targets the Ai9 and related transgenes