149 results for AAV

AAV

Delivery System  - [In Vivo] [AAV tropism, Delivery Systems Initiative] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing name -->  AAV  -->  AAV tropism  -->  AAV  -->  AAV  -->  AAV3b-ZsGreen-Cre  AAVrh8-ZsGreen-Cre  AAV9-CMV-Cre  AAV6-ZsGreen-Cre  AAV4-ZsGreen-Cre  -->  AAV name -->  AAV  -->  AAV Tropism project
See vector details

AAV Tropism project

Experiment  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice name -->  AAV Tropism project  -->  AAV tropism  -->  AAV  -->  AAV Tropism project  -->  AAV6-ZsGreen-Cre  AAV3b-ZsGreen-Cre  AAV4-ZsGreen-Cre  AAV8-ZsGreen-Cre  AAV5-ZsGreen-Cre  -->  Ten AAV serotypes delivering Cre recombinase were tested by intravenous delivery into Ai9 mice and chacterized  -->  AAV name -->  AAV Tropism project  -->  AAV Tropism project
Lutz Cathleen M , Gao Guang-Ping , Heaney Jason D , Murray Stephen A , Lagor William Raymond , Dickinson Mary E  Last Updated Date: 2023-02-10
 
Ten AAV serotypes delivering Cre recombinase were tested by intravenous delivery into Ai9 mice and chacterized for biodistribution across 20 tissues by quantitative PCR and imaging

Conklin_Fast-Seq AAV Protocol

Protocol 
Matched on:  -->  Conklin_Fast-Seq AAV Protocol  -->  Published procedure for AAV composition measurement using fast-seq. Maynard et al. name -->  Conklin_Fast-Seq AAV Protocol
Published procedure for AAV composition measurement using fast-seq. Maynard et al. Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings. Hum Gene Ther Methods . 2019 Dec;30(6):195-205. doi: 10.1089/hgtb.2019.110.

Heaney-SATC_Gao-Validation_Intratracheal Delivery of AAV in Mice

Protocol  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  Heaney-SATC_Gao-Validation_Intratracheal Delivery of AAV in Mice  -->  Procedure for Intratracheal (IT) delivery of AAV in mouse lung.  -->  AAV name -->  Heaney-SATC_Gao-Validation_Intratracheal Delivery of AAV in Mice
Procedure for Intratracheal (IT) delivery of AAV in mouse lung.

SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice

Project  [AAV tropism]
Matched on:  -->  AAV tropism  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice name -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice
Lutz Cathleen M , Gao Guang-Ping , Heaney Jason D , Murray Stephen A , Lagor William Raymond , Dickinson Mary E  Last Updated Date: 2023-02-10
 
Show Experiments (1)

AAV Tropism project

Evolving High Potency AAV Vectors for Neuromuscular Genome Editing

Project  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  Recombinant adeno-associated viruses (AAV) have emerged as safe and effective vectors for clinical gene  The current proposal is on a comprehensive and innovative approach to evolve high potency AAV variants name -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Asokan Aravind  Last Updated Date: 2020-11-19 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.
Show Experiments (7)

Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter)
Cre Recombinase dose escalation study in Ai9 mice
Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.

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

Experiment  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular  -->  AAVcc47-Cre  AAVcc47-SaCas9-Ai9  -->  Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery.  -->  AAV name -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular  -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular
Heaney Jason D  Last Updated Date: 2021-03-30
 
Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues.

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_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_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_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_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_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_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_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_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_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_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_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_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

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

AAV6-ZsGreen-Cre

Vector  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  -->  AAV tropism  -->  AAV symbol -->  AAV6-ZsGreen-Cre  -->  AAV6-ZsGreen-Cre  -->  AAV backbone with a bi-directional promoter driving zsGreen and Cre  -->  AAV  -->  AAV Tropism project
AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

AAV9-Ai9-sgRNA2-CB-SaCas9

Vector  - [In Vivo] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAV9-Ai9-sgRNA2-CB-SaCas9  -->  AAV9-Ai9-sgRNA2-CB-SaCas9  AAV9-Ai9-sgRNA1-CB-SaCas9  -->  AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus  -->  AAV
AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus

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

AAV9-ZsGreen-Cre

Vector  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  -->  AAV tropism  -->  AAV symbol -->  AAV9-ZsGreen-Cre  -->  AAV9-ZsGreen-Cre  -->  AAV backbone with a bi-directional promoter driving zsGreen and Cre  -->  AAV  -->  AAV Tropism project
AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

AAVcc47-CMV-SaCas9

Vector  - [In Vivo] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc47-CMV-SaCas9  -->  AAVcc47-Ai9-sgRNA1 + sgRNA2  AAVcc47-CMV-SaCas9  -->  AAV
AAVcc47 delivering CMV driven SaCas9

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

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-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-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

AAV7-ZsGreen-Cre

Vector  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  -->  AAV tropism  -->  AAV symbol -->  AAV7-ZsGreen-Cre  -->  AAV7-ZsGreen-Cre  -->  AAV backbone with a bi-directional promoter driving zsGreen and Cre  -->  AAV  -->  AAV Tropism project
AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

AAV8-ZsGreen-Cre

Vector  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  -->  AAV tropism  -->  AAV symbol -->  AAV8-ZsGreen-Cre  -->  AAV8-ZsGreen-Cre  -->  AAV backbone with a bi-directional promoter driving zsGreen and Cre  -->  AAV  -->  AAV Tropism project
AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

AAVcc47-Ai9-sgRNA1-CB-SaCas9

Vector  - [In Vivo] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc47-Ai9-sgRNA1-CB-SaCas9  -->  AAVcc47-Ai9-sgRNA2-CB-SaCas9  AAVcc47-Ai9-sgRNA1-CB-SaCas9  -->  AAV
AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus

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-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-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

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-Cre

Vector  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on: symbol -->  AAVcc47-Cre  -->  AAVcc47-Cre  -->  AAV  -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular
AAV2/5 expressing Cre recombinase

AAVcc47-SaCas9-Ai9

Vector  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on: symbol -->  AAVcc47-SaCas9-Ai9  -->  AAVcc47-SaCas9-Ai9  -->  AAV  -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular
AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter

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-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-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

AAV5-ZsGreen-Cre

Vector  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  -->  AAV tropism  -->  AAV symbol -->  AAV5-ZsGreen-Cre  -->  AAV5-ZsGreen-Cre  -->  AAV backbone with a bi-directional promoter driving zsGreen and Cre  -->  AAV  -->  AAV Tropism project
AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

AAV9-Ai9-sgRNA1 + sgRNA2

Vector  - [In Vivo] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAV9-Ai9-sgRNA1 + sgRNA2  -->  AAV9-Ai9-sgRNA1 + sgRNA2  AAV9-CMV-SaCas9  -->  AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus  -->  AAV
AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus

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

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

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

AAVrh10-ZsGreen-Cre

Vector  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  -->  AAV tropism  -->  AAV symbol -->  AAVrh10-ZsGreen-Cre  -->  AAVrh10-ZsGreen-Cre  -->  AAV backbone with a bi-directional promoter driving zsGreen and Cre  -->  AAV  -->  AAV Tropism project
AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

AAVrh74-ZsGreen-Cre

Vector  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  -->  AAV tropism  -->  AAV symbol -->  AAVrh74-ZsGreen-Cre  -->  AAVrh74-ZsGreen-Cre  -->  AAV backbone with a bi-directional promoter driving zsGreen and Cre  -->  AAV  -->  AAV Tropism project
AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

AAVrh8-ZsGreen-Cre

Vector  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  -->  AAV tropism  -->  AAV symbol -->  AAVrh8-ZsGreen-Cre  -->  AAVrh8-ZsGreen-Cre  -->  AAV backbone with a bi-directional promoter driving zsGreen and Cre  -->  AAV  -->  AAV Tropism project
AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

AAV4-ZsGreen-Cre

Vector  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  -->  AAV tropism  -->  AAV symbol -->  AAV4-ZsGreen-Cre  -->  AAV4-ZsGreen-Cre  -->  AAV backbone with a bi-directional promoter driving zsGreen and Cre  -->  AAV  -->  AAV Tropism project
AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

AAV9-GFP

Vector  - [In Vivo] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAV9-GFP  -->  AAV9-GFP  -->  AAV
AAV2/9 self complementary vector expressing GFP driven by CBh promoter

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

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-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-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

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

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

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

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-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

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.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

AAV3b-ZsGreen-Cre

Vector  - [In Vivo] [AAV tropism] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  -->  AAV tropism  -->  AAV symbol -->  AAV3b-ZsGreen-Cre  -->  AAV3b-ZsGreen-Cre  -->  AAV backbone with a bi-directional promoter driving zsGreen and Cre  -->  AAV  -->  AAV Tropism project
AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2

Vector  - [In Vitro] [Delivery Systems Initiative] [Mouse]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA

Tsai_T Cell Transfection Protocol

Protocol  - [In Vitro] [Biological Systems] [Human]
Matched on:  -->  AAVS1_site_14  AAVS1_site_13  AAVS1_site_12  AAVS1_site_11  AAVS1_site_10  -->  AAVS1
Procedure for T cell transfection.

demo VV03-Cre

Vector  [Mouse]
Matched on:  -->  AAV
DEMO viral vector for demo purpose

demo VV02-Cre

Vector  [Mouse]
Matched on:  -->  AAV
DEMO viral vector for demo purpose

Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)

Experiment  - [In Vivo] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV9-Ai9-sgRNA1 + sgRNA2  AAVcc47-Ai9-sgRNA1 + sgRNA2  AAVcc47-CMV-SaCas9  AAV9-CMV-SaCas9  -->  single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV  -->  AAV name -->  Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9
Asokan Aravind  Last Updated Date: 2021-09-16
 
A dual vector strategy was employed: one delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV cassettes). This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=5) by intravenous injection in Ai9 mice. A total dose of 4e12vg was injected into each mouse and vectors mixed in a 1:4 ratio of cas9 to guide RNA (1e12vg of CMV Sacas9 vector and 3e12vg of the sgRNA vector) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart.

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

Experiment  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing  -->  AAV name -->  Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to  -->  AAV BI28 (novel engineered variant)
Heaney Jason D  Last Updated Date: 2023-05-10
 
Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing. Editing detected and quantified in brain by generation of tdTomato fluorescent protein signal from Ai9 reporter mice

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.

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

Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.

Experiment  - [In Vivo] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV9-mCherry  AAVcc47-mCherry  -->  AAV
Asokan Aravind  Last Updated Date: 2020-11-19
 
C57BL/6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding an mCherry reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification.

Cre recombinase

Genome Editor  - [In Vivo, In Vitro] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse]
Matched on:  -->  AAVcc47-Cre  -->  Cre recombinase delivered by AAV (see vector details)  -->  AAV  -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular
Cre recombinase delivered by AAV (see vector details)

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.

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

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

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.

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)

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.

demo VV01-Cre

Vector  [Mouse]
Matched on:  -->  AAV
DEMO viral vector for demo purpose

demo VV05-Cre

Vector  [Mouse]
Matched on:  -->  AAV
DEMO viral vector for demo purpose

BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2

Vector  - [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse]
Matched on:  -->  Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP  -->  AAV  -->  AAV BI28 (novel engineered variant)
Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds

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

demo VV04-Cre

Vector  [Mouse]
Matched on:  -->  AAV
DEMO viral vector for demo purpose

Ai9 mouse

Model System  - [In Vivo] [Small Animal Testing Center (SATC), AAV tropism, Delivery Systems Initiative] [Mouse]
Matched on:  -->  SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV tropism  -->  AAV  -->  AAVrh8-ZsGreen-Cre  AAVcc47-CMV-SaCas9  AAV9-CMV-SaCas9  AAV6-ZsGreen-Cre  AAV9-CMV-Cre  -->  AAV  -->  AAV BI28 (novel engineered variant)  -->  AAV Tropism project
Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.
Show Experiments (10)

Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
Testing AAV5 for activation of tdTomato in mouse airway
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter)
AAV Tropism project
Cre Recombinase dose escalation study in Ai9 mice
Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.

Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter)

Experiment  - [In Vivo] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV9-Ai9-sgRNA1 + sgRNA2  AAVcc47-Ai9-sgRNA1 + sgRNA2  AAVcc47-CMV-SaCas9  AAV9-CMV-SaCas9  -->  single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV  -->  AAV name -->  Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to
Asokan Aravind  Last Updated Date: 2021-09-16
 
A dual vector strategy was employed: one delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV cassettes). This strategy was evaluted with both AAV9 (n=3) and AAVcc47 (n=3) by intravenous injection in Ai9 mice. A total dose of 3e12vg was injected into each mouse (1.5e12vg each vector mixed 1:1) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart.

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.

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.

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.

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

Ai9 SaCas9 Guide A

Guide  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAVcc47-SaCas9-Ai9  -->  AAV  -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular
This gRNA targets the Ai9 and related transgenes

Ai9 SaCas9 Guide B

Guide  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAVcc47-SaCas9-Ai9  -->  AAV  -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular
This gRNA targets the Ai9 and related transgenes

SauCas9

Genome Editor  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAVcc47-SaCas9-Ai9  -->  AAV  -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular

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.

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

Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.

Experiment  - [In Vivo] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAVcc84-GFP  AAVcc81-GFP  AAV9-GFP  -->  AAV
Asokan Aravind  Last Updated Date: 2020-11-19
 
C57BL/6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding a GFP reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification.

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)

Deverman_Area Based Quantification of Editing Efficiency Protocol

Protocol  - [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  -->  AAV  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  -->  AAV BI28 (novel engineered variant)
Procedure for non-IHC based image quantification of editing.

Deverman_Comprehensive Methods

Protocol  - [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  -->  Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue  -->  AAV  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  -->  AAV BI28 (novel engineered variant)
Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue processing and IHC.

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

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

Deverman_AAV Production and Administration Protocol

Protocol  - [In Vitro] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  -->  Published procedures for AAV production, delivery, and tissue imaging. Challis et al.  Systemic AAV vectors for widespread and targeted gene delivery in rodents.  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb
Published procedures for AAV production, delivery, and tissue imaging. Challis et al. Systemic AAV vectors for widespread and targeted gene delivery in rodents. Nat Protoc. 2019 Feb;14(2):379-414. doi: 10.1038/s41596-018-0097-3.

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

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

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

Chaikof-Associated Protocol 2_Off-target editing AND Primers for sequencing analysis

Protocol  - [In Vivo] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  AAV-Pcsk9  -->  compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV
This protocol describes in vivo adminstration and subsequent analysis of off-target editing in the liver.

R1-unmodified

Guide  - [In Vitro] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb
This gRNA targets the Ai9 and related transgenes

R2-unmodified

Guide  - [In Vitro] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb
This gRNA targets the Ai9 and related transgenes

Ai9 mouse (BCM)

Model System  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAVcc47-Cre  AAVcc47-SaCas9-Ai9  -->  AAV  -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular
Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.

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

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

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

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

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

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

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

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

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

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

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

Heaney_SATC Tissue Processing, Imaging and Analysis

Protocol  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAVcc47-Cre  AAVcc47-SaCas9-Ai9  -->  AAV  -->  AAV BI28 (novel engineered variant)  -->  Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular
Procedure for tissue preparation, imaging and analysis.

HEK-293T with Ai9 transient reporter assay

Model System  - [In Vitro] [Delivery Systems Initiative] [Human]
Matched on:  -->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)
HEK-293T cells transfected with an Ai9 inducible transgene reporter plasmid used to test gene editing activity by fluorescence. HEK293T is an epithelial-like cell that was isolated from the kidney of a patient.

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

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

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

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

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

R2-modified

Guide  - [In Vivo, In Vitro] [Delivery Systems Initiative] [Mouse]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1  -->  AAV  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb  -->  AAV BI28 (novel engineered variant)
This gRNA targets the Ai9 and related transgenes

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

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

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

ssAAV5-sgB.saCas9

Vector  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV

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

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

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

Experiment  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV
Heaney Jason D  Last Updated Date: 2021-03-30
 
AAV5 encoding CRISPR/Cas editing machinery were delivered to the lungs of reporter mice by intratracheal instillation. After 4 weeks incubation, the mice were dissected and the lungs imaged for the presence of tdTomato fluorescence, indicating successful editing. Editing calculated by dividing the number of tdTomato+ red cells by the number of nuclei in each airway

BCM_ssAAV5-Sa_sgB

Guide  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV
This gRNA targets the Ai9 and related transgenes

SaCas9

Genome Editor  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV

scAAV5-Cre-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

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.

BCM_ssAAV5-Sp_sgA

Guide  - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse]
Matched on:  -->  AAV
This sgRNA targets the Ai9 and related transgenes

ssAAV5-sgA+sgB-U1A.GFP

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

149 results for AAV

Category Type Name Description Last Updated Date View Associated..
Delivery System Viral vector AAV See vector details
Experiment In Vivo AAV Tropism project Ten AAV serotypes delivering Cre recombinase were tested by intravenous delivery into Ai9 mice and chacterized for biodistribution across 20 tissues by quantitative PCR and imaging 2023-02-10
Protocol Conklin_Fast-Seq AAV Protocol Published procedure for AAV composition measurement using fast-seq. Maynard et al. Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings. Hum Gene Ther Methods . 2019 Dec;30(6):195-205. doi: 10.1089/hgtb.2019.110.
Protocol Heaney-SATC_Gao-Validation_Intratracheal Delivery of AAV in Mice Procedure for Intratracheal (IT) delivery of AAV in mouse lung.
Project SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice 2023-02-10
Show Experiments (1)

AAV Tropism project

Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Recombinant adeno-associated viruses (AAV) have emerged as safe and effective vectors for clinical gene therapy applications including systemic treatment of neuromuscular diseases such as Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD), and Giant Axonal Neuropathy (GAN) amongst others. However, genome editing in neuromuscular tissue, in particular, is challenging. The current proposal is on a comprehensive and innovative approach to evolve high potency AAV variants for systemic neuromuscular genome editing. 2020-11-19
Show Experiments (7)

Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter)
Cre Recombinase dose escalation study in Ai9 mice
Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.

Experiment In Vivo Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues. 2021-03-30
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_12 Targets AAVS1 safe harbor locus
Guide AAVS1_site_13 Targets AAVS1 safe harbor locus
Guide AAVS1_site_04 Targets AAVS1 safe harbor locus
Guide AAVS1_site_11 Targets AAVS1 safe harbor locus
Guide AAVS1_site_01 Targets AAVS1 safe harbor locus
Guide AAVS1_site_05 Targets AAVS1 safe harbor locus
Guide AAVS1_site_06 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_03 Targets AAVS1 safe harbor locus
Guide AAVS1_site_09 Targets AAVS1 safe harbor locus
Guide AAVS1_site_02 Targets AAVS1 safe harbor locus
Guide AAVS1_site_07 Targets AAVS1 safe harbor locus
Guide AAVS1_site_14 Targets AAVS1 safe harbor locus
Vector Viral Vector AAV6-ZsGreen-Cre AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

Vector Viral Vector AAV9-Ai9-sgRNA2-CB-SaCas9 AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus
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 AAV9-ZsGreen-Cre AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

Vector Viral Vector AAVcc47-CMV-SaCas9 AAVcc47 delivering CMV driven SaCas9
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 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-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-LoxP2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
Vector Viral Vector AAV7-ZsGreen-Cre AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

Vector Viral Vector AAV8-ZsGreen-Cre AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

Vector Viral Vector AAVcc47-Ai9-sgRNA1-CB-SaCas9 AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
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-Pcsk9
Vector Viral Vector AAV9-Ai9-sgRNA1-CB-SaCas9 AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAV9-CMV-SaCas9 AAV serotype 9 delivering CMV driven SaCas9
Vector Viral Vector AAVcc47-Cre AAV2/5 expressing Cre recombinase
Vector Viral Vector AAVcc47-SaCas9-Ai9 AAV2/9 expressing SaCas9 and single sgRNA under U6 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-gRNA-LoxP-Sa-L1 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
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 AAV5-ZsGreen-Cre AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

Vector Viral Vector AAV9-Ai9-sgRNA1 + sgRNA2 AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus
Vector Viral Vector AAVcc47-Ai9-sgRNA2-CB-SaCas9 AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAVcc47-CMV-Cre AAVcc47 delivering CMV Cre Recombinase
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 AAVrh10-ZsGreen-Cre AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

Vector Viral Vector AAVrh74-ZsGreen-Cre AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

Vector Viral Vector AAVrh8-ZsGreen-Cre AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

Vector Viral Vector AAV4-ZsGreen-Cre AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

Vector Viral Vector AAV9-GFP AAV2/9 self complementary vector expressing GFP driven by CBh promoter
Vector Viral Vector AAV9-mCherry AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter
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-LoxP1 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
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 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
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 AAVcc47-Ai9-sgRNA1 + sgRNA2 AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus
Vector Viral Vector AAV9-CMV-Cre AAV serotype 9 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 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.pU1a-SpCas9 Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS
Vector Viral Vector AAV3b-ZsGreen-Cre AAV backbone with a bi-directional promoter driving zsGreen and Cre
Show Experiments (1)

AAV Tropism project

Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
Protocol Tsai_T Cell Transfection Protocol Procedure for T cell transfection.
Vector Viral Vector demo VV03-Cre DEMO viral vector for demo purpose
Vector Viral Vector demo VV02-Cre DEMO viral vector for demo purpose
Experiment In Vivo Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) A dual vector strategy was employed: one delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV cassettes). This strategy was evaluted with both AAV9 (n=4) and AAVcc47 (n=5) by intravenous injection in Ai9 mice. A total dose of 4e12vg was injected into each mouse and vectors mixed in a 1:4 ratio of cas9 to guide RNA (1e12vg of CMV Sacas9 vector and 3e12vg of the sgRNA vector) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. 2021-09-16
Experiment In Vivo Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing. Editing detected and quantified in brain by generation of tdTomato fluorescent protein signal from Ai9 reporter mice 2023-05-10
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
Genome Editor Cas Nuclease SaCas9-Lagor
Experiment In Vivo Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. C57BL/6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding an mCherry reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification. 2020-11-19
Genome Editor Recombinase Cre recombinase Cre recombinase delivered by AAV (see vector details)
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
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 Cas Nuclease SpCas9 HA-SV40NLS-SpCas9-SV40NLS
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
Genome Editor Base Editor (SpCas9) TadA-8e V106W Catalytically impaired Cas fused to evolved TadA deaminase (TadA-8e V106W)
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
Vector Viral Vector demo VV01-Cre DEMO viral vector for demo purpose
Vector Viral Vector demo VV05-Cre DEMO viral vector for demo purpose
Vector Viral Vector BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2 Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds
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
Vector Viral Vector demo VV04-Cre DEMO viral vector for demo purpose
Model System Animal Ai9 mouse Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.
Show Experiments (10)

Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)
Testing AAV5 for activation of tdTomato in mouse airway
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter)
AAV Tropism project
Cre Recombinase dose escalation study in Ai9 mice
Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.

Experiment In Vivo Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) A dual vector strategy was employed: one delivering two single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV cassettes). This strategy was evaluted with both AAV9 (n=3) and AAVcc47 (n=3) by intravenous injection in Ai9 mice. A total dose of 3e12vg was injected into each mouse (1.5e12vg each vector mixed 1:1) and organs were harvested 4 weeks post injection. Editing efficency was determined by calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. 2021-09-16
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
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 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
Model System Animal C57BL/6 mouse (Asokan study)
Guide Ai9 SaCas9 Guide A This gRNA targets the Ai9 and related transgenes
Guide Ai9 SaCas9 Guide B This gRNA targets the Ai9 and related transgenes
Genome Editor Cas Nuclease SauCas9
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
Guide L1-unmodified This gRNA targets the Ai9 and related transgenes
Experiment In Vivo Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. C57BL/6 mice (N=3) were injected intravenously at a dose of 5e13 vg/kg per mouse with a self-complementary AAV9 or ccAAV vector encoding a GFP reporter. The biodistribution of of virus transduction was chacterized in various tissues and cell types by fluorescence imaging quantification. 2020-11-19
Genome Editor Cas Nuclease