90 results for aav

AAVS1_site_01   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_01 grnaLabId ->  AAVS1_site_01 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_07   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_07 grnaLabId ->  AAVS1_site_07 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_09   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_09 grnaLabId ->  AAVS1_site_09 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_14   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_14 grnaLabId ->  AAVS1_site_14 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_04   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_04 grnaLabId ->  AAVS1_site_04 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_08   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_08 grnaLabId ->  AAVS1_site_08 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_05   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_05 grnaLabId ->  AAVS1_site_05 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_06   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_06 grnaLabId ->  AAVS1_site_06 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_10   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_10 grnaLabId ->  AAVS1_site_10 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_11   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_11 grnaLabId ->  AAVS1_site_11 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_02   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_02 grnaLabId ->  AAVS1_site_02 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_03   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_03 grnaLabId ->  AAVS1_site_03 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_13   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_13 grnaLabId ->  AAVS1_site_13 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAVS1_site_12   - Guide - [In Vitro]
Matched on: Name ->  AAVS1_site_12 grnaLabId ->  AAVS1_site_12 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Targets AAVS1 safe harbor locus
View Associated: 
AAV9-CMV-Cre   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-CMV-Cre Name ->  AAV9-CMV-Cre vectorName ->  AAV9-CMV-Cre description ->  AAV serotype 9 delivering CMV Cre Recombinase vectorSubtype ->  AAV generatedDescription ->  ........................................AAV serotype 9 delivering CMV Cre Recombinase....CAG-loxP-stop-loxP-tdtomato
AAV serotype 9 delivering CMV Cre Recombinase
View Associated: 
AAV9-GFP   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-GFP Name ->  AAV9-GFP vectorName ->  AAV9-GFP vectorSubtype ->  AAV
AAV2/9 self complementary vector expressing GFP driven by CBh promoter
View Associated: 
AAVcc47-Cre   - Vector
Matched on: Name ->  AAVcc47-Cre Name ->  AAVcc47-Cre vectorName ->  AAVcc47-Cre vectorSubtype ->  AAV experimentName ->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
AAV2/5 expressing Cre recombinase
View Associated: 
AAVcc81-GFP   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAVcc81-GFP Name ->  AAVcc81-GFP vectorName ->  AAVcc81-GFP vectorSubtype ->  AAV
AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter
View Associated: 
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 vectorName ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA generatedDescription ->  ........This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven ; SaCas9 and U6 promoter driven gRNA..AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter vectorAnnotatedMap ->  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
View Associated: 
AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 vectorName ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold generatedDescription ->  ....This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven SaCas9 vectorAnnotatedMap ->  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
View Associated: 
AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2 Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2 vectorName ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold generatedDescription ->  ..This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter vectorAnnotatedMap ->  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
View Associated: 
AAV.pU1a-SpCas9   - Vector
Matched on: Name ->  AAV.pU1a-SpCas9 Name ->  AAV.pU1a-SpCas9 ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  AAV.pU1a-SpCas9 ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorSubtype ->  AAV
Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS
View Associated: 
AAV9-CMV-SaCas9   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-CMV-SaCas9 Name ->  AAV9_pTR_self comp 2xU6-Ai9 guides ; AAV9-Ai9-sgRNA1 + sgRNA2 ; AAV9-CMV-SaCas9AAVcc47_pTR_self comp 2xU6-Ai9 guides vectorName ->  AAV9_pTR_self comp 2xU6-Ai9 guides ; AAV9-Ai9-sgRNA1 + sgRNA2 ; AAV9-CMV-SaCas9AAVcc47_pTR_self comp 2xU6-Ai9 guides description ->  AAV serotype 9 delivering CMV driven SaCas9 vectorSubtype ->  AAV generatedDescription ->  ..................................AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting ; the Ai9 locus..AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector ; ) targeting Ai9 transgene..AAV serotype 9 delivering CMV driven SaCas9..AAVcc47 delivering u6 promoter
AAV serotype 9 delivering CMV driven SaCas9
View Associated: 
AAVcc47-mCherry   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAVcc47-mCherry Name ->  AAVcc47-mCherry vectorName ->  AAVcc47-mCherry vectorSubtype ->  AAV
AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter
View Associated: 
AAVcc47-SaCas9-Ai9   - Vector
Matched on: Name ->  AAVcc47-SaCas9-Ai9 Name ->  AAVcc47-SaCas9-Ai9 vectorName ->  AAVcc47-SaCas9-Ai9 vectorSubtype ->  AAV experimentName ->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter
View Associated: 
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 vectorName ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA generatedDescription ->  ......This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven vectorAnnotatedMap ->  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
View Associated: 
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 vectorName ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA generatedDescription ->  ........This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven vectorAnnotatedMap ->  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
View Associated: 
AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 vectorName ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold generatedDescription ->  ......This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven vectorAnnotatedMap ->  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
View Associated: 
AAV9-Ai9-sgRNA1 + sgRNA2   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-Ai9-sgRNA1 + sgRNA2 Name ->  AAV9-Ai9-sgRNA1 + sgRNA2 ; AAV9-CMV-SaCas9 vectorName ->  AAV9-Ai9-sgRNA1 + sgRNA2 ; AAV9-CMV-SaCas9 description ->  AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus vectorSubtype ->  AAV generatedDescription ->  ..................AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus ; ..AAV serotype 9 delivering CMV driven SaCas9....CAG-loxP-stop-loxP-tdtomato reporter cassette which
AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus
View Associated: 
AAVcc47-CMV-SaCas9   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAVcc47-CMV-SaCas9 Name ->  AAVcc47-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9 vectorName ->  AAVcc47-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9 vectorSubtype ->  AAV generatedDescription ->  ..............AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus.
AAVcc47 delivering CMV driven SaCas9
View Associated: 
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 vectorName ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA generatedDescription ->  ......This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven vectorAnnotatedMap ->  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
View Associated: 
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1 Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1 vectorName ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA generatedDescription ->  ..This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter vectorAnnotatedMap ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
View Associated: 
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 vectorName ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA generatedDescription ->  ........This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven vectorAnnotatedMap ->  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
View Associated: 
AAVcc47_pTR_self comp 2xU6-Ai9 guides   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAVcc47_pTR_self comp 2xU6-Ai9 guides Name ->  AAV9-CMV-SaCas9AAVcc47_pTR_self comp 2xU6-Ai9 guides vectorName ->  AAV9-CMV-SaCas9AAVcc47_pTR_self comp 2xU6-Ai9 guides vectorSubtype ->  AAV generatedDescription ->  ..........AAV serotype 9 delivering CMV driven SaCas9..AAVcc47 delivering u6 promoter driving sgRNA 1
AAVcc47 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene
View Associated: 
AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 vectorName ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold generatedDescription ->  ......This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven vectorAnnotatedMap ->  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
View Associated: 
AAV9-Ai9-sgRNA1-CB-SaCas9   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-Ai9-sgRNA1-CB-SaCas9 Name ->  AAV9-Ai9-sgRNA2-CB-SaCas9AAV9-Ai9-sgRNA1-CB-SaCas9 vectorName ->  AAV9-Ai9-sgRNA2-CB-SaCas9AAV9-Ai9-sgRNA1-CB-SaCas9 description ->  AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus vectorSubtype ->  AAV generatedDescription ->  ..........AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus..AAV serotype 9 delivering
AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
View Associated: 
AAV9-Ai9-sgRNA2-CB-SaCas9   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-Ai9-sgRNA2-CB-SaCas9 Name ->  AAV9-Ai9-sgRNA2-CB-SaCas9AAV9-Ai9-sgRNA1-CB-SaCas9 vectorName ->  AAV9-Ai9-sgRNA2-CB-SaCas9AAV9-Ai9-sgRNA1-CB-SaCas9 description ->  AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus vectorSubtype ->  AAV generatedDescription ->  ..........AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus..AAV serotype 9 delivering
AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus
View Associated: 
AAVcc47-Ai9-sgRNA1-CB-SaCas9   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAVcc47-Ai9-sgRNA1-CB-SaCas9 Name ->  AAVcc47-Ai9-sgRNA2-CB-SaCas9AAVcc47-Ai9-sgRNA1-CB-SaCas9 vectorName ->  AAVcc47-Ai9-sgRNA2-CB-SaCas9AAVcc47-Ai9-sgRNA1-CB-SaCas9 vectorSubtype ->  AAV
AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
View Associated: 
AAVcc47-Ai9-sgRNA1 + sgRNA2   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAVcc47-Ai9-sgRNA1 + sgRNA2 Name ->  AAVcc47-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9 vectorName ->  AAVcc47-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9 description ->  AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus vectorSubtype ->  AAV generatedDescription ->  ..............AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus.
AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus
View Associated: 
AAVcc47-CMV-Cre   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAVcc47-CMV-Cre Name ->  AAVcc47-CMV-Cre vectorName ->  AAVcc47-CMV-Cre vectorSubtype ->  AAV
AAVcc47 delivering CMV Cre Recombinase
View Associated: 
AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 vectorName ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 ; AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold generatedDescription ->  ........This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven ; SaCas9 and U6 promoter driven gRNA..AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter vectorAnnotatedMap ->  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
View Associated: 
AAV9_pTR_self comp 2xU6-Ai9 guides   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9_pTR_self comp 2xU6-Ai9 guides Name ->  AAV9_pTR_self comp 2xU6-Ai9 guides ; AAV9-CMV-SaCas9 vectorName ->  AAV9_pTR_self comp 2xU6-Ai9 guides ; AAV9-CMV-SaCas9 description ->  AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting vectorSubtype ->  AAV generatedDescription ->  ..........AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) ; targeting Ai9 transgene..AAV serotype 9 delivering CMV driven SaCas9....CAG-loxP-stop-loxP-tdtomato reporter
AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene
View Associated: 
AAV9-mCherry   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-mCherry Name ->  AAV9-mCherry vectorName ->  AAV9-mCherry vectorSubtype ->  AAV
AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter
View Associated: 
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 Name ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 vectorName ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA generatedDescription ->  ..This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter vectorAnnotatedMap ->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
View Associated: 
AAVcc47-Ai9-sgRNA2-CB-SaCas9   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAVcc47-Ai9-sgRNA2-CB-SaCas9 Name ->  AAVcc47-Ai9-sgRNA2-CB-SaCas9AAVcc47-Ai9-sgRNA1-CB-SaCas9 vectorName ->  AAVcc47-Ai9-sgRNA2-CB-SaCas9AAVcc47-Ai9-sgRNA1-CB-SaCas9 vectorSubtype ->  AAV
AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus
View Associated: 
AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1 Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1 vectorName ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold generatedDescription ->  ..This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter vectorAnnotatedMap ->  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
View Associated: 
AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1   - Vector
Matched on: Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 Name ->  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 vectorName ->  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 description ->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold generatedDescription ->  ........This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven vectorAnnotatedMap ->  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
View Associated: 
AAVcc84-GFP   - Vector
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAVcc84-GFP Name ->  AAVcc84-GFP vectorName ->  AAVcc84-GFP vectorSubtype ->  AAV
AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter
View Associated: 
Evolving High Potency AAV Vectors for Neuromuscular Genome Editing   - Study
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Asokan Aravind   Date Of Submission: 2021-09-16
View Associated: 
Evolving High Potency AAV Vectors for Neuromuscular Genome Editing   - Study
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Asokan Aravind   Date Of Submission: 2020-11-19
View Associated: 
[] Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.   - Experiment - [In Vivo]
Matched on: Name ->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing vectorName ->  AAVcc47-CreAAVcc47-SaCas9-Ai9 description ->  Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. vectorSubtype ->  AAV generatedDescription ->  Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. experimentName ->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Heaney Jason D
Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues.
View Associated: 
CD4/CD8 Human Primary T cell   - Model System - [In Vitro]
Matched on: grnaLabId ->  AAVS1_site_14AAVS1_site_13AAVS1_site_12AAVS1_site_11AAVS1_site_10 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
CD4/CD8 Human Primary T cell
View Associated: 
P3 Nucleofection Kit   - Delivery System - [In Vitro]
Matched on: grnaLabId ->  AAVS1_site_14AAVS1_site_13AAVS1_site_12AAVS1_site_11AAVS1_site_10 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Nuceleofection kit to be used with Lonza's nucleofection system.
View Associated: 
Ai9 mouse   - Model System - [In Vivo]
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-CMV-CreAAVcc47-CMV-SaCas9AAV9-CMV-SaCas9AAV.pU1a-SpCas9AAVcc47-CMV-Cre vectorName ->  AAV9-CMV-CreAAVcc47-CMV-SaCas9AAV9-CMV-SaCas9AAV.pU1a-SpCas9AAVcc47-CMV-Cre vectorSubtype ->  AAV generatedDescription ->  This sgRNA targets the Ai9 and related transgenes ..This gRNA targets the Ai9 and related transgenes ..AAV ; 1 + sgRNA2 targeting the Ai9 locus..AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus..AAV ; serotype 9 delivering CMV Cre Recombinase..Novel engineered AAV BI28 variant expressing S. aureus Cas9 ; HA-SV40NLS-SpCas9-SV40NLS..AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus..AAV ; serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus..Novel engineered AAV BI28 variant capsidSerotype ->  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.
View Associated: 
A novel human T cell platform to define biological adverse effects of genome editing   - Experiment - [In Vitro]
Matched on: grnaLabId ->  AAVS1_site_14AAVS1_site_13AAVS1_site_12AAVS1_site_11AAVS1_site_10 Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
Tsai Shengdar Q
110 guide RNAs and SpCas9 were transfected into human T-cells. Indel rates were measured by targeted amplicon deep sequencing.
View Associated: 
SauCas9   - Genome Editor
Matched on: Name ->  AAVcc47-SaCas9-Ai9 vectorName ->  AAVcc47-SaCas9-Ai9 vectorSubtype ->  AAV experimentName ->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
View Associated: 
SpCas9   - Genome Editor
Matched on: grnaLabId ->  AAVS1_site_14AAVS1_site_13AAVS1_site_12AAVS1_site_11AAVS1_site_10 Name ->  AAV.pU1a-SpCas9 ; PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  AAV.pU1a-SpCas9 ; PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorSubtype ->  AAV Guide Target Locus ->  AAVS1 guideTargetLocus ->  AAVS1
HA-SV40NLS-SpCas9-SV40NLS
View Associated: 
SaCas9   - Genome Editor
Matched on: Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 vectorName ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 vectorSubtype ->  AAV generatedDescription ->  the mTmG, Ai9 and related transgenes at two sites..This gRNA targets the Ai9 and related transgenes ..AAV ; shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA..AAV shuttle vector with ; promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence..Novel engineered AAV ; fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds..Novel engineered AAV vectorAnnotatedMap ->  AAV-CMV-SaCas9-U6-modified scaffold-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-R1.gb ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb capsidSerotype ->  AAV BI28 (novel engineered variant)
View Associated: 
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]
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9AAV9-CMV-SaCas9 vectorName ->  AAV9-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9AAV9-CMV-SaCas9 description ->  single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV vectorSubtype ->  AAV generatedDescription ->  single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV ; calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. ........................AAV ; u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus..AAVcc47 delivering CMV driven SaCas9..AAV ; fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus...........................AAV ; u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus..AAVcc47 delivering CMV driven SaCas9..AAV
Asokan Aravind
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.
View Associated: 
[] Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain   - Experiment - [In Vivo]
Matched on: description ->  Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing vectorSubtype ->  AAV generatedDescription ->  Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing ; ................................This gRNA targets the Ai9 and related transgenes ..Novel engineered AAV ; fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds..Novel engineered AAV ; ................................This gRNA targets the Ai9 and related transgenes ..Novel engineered AAV ; fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds..Novel engineered AAV Name ->  Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to capsidSerotype ->  AAV BI28 (novel engineered variant)
Heaney Jason D
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
View Associated: 
Testing AAV5 for activation of tdTomato in HEK293T cells   - Experiment - [In Vitro]
Matched on: Name ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) description ->  AAV shuttle plasmids expressing SpCas9 and guide RNAs targeting the Ai9 transgene were tested in HEK293T generatedDescription ->  AAV shuttle plasmids expressing SpCas9 and guide RNAs targeting the Ai9 transgene were tested in HEK293T
Gao Guang-Ping
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.
View Associated: 
Testing AAV5 for activation of tdTomato in mouse airway   - Experiment - [In Vivo]
Matched on: Name ->  AAV.pU1a-SpCas9 ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  AAV.pU1a-SpCas9 ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorSubtype ->  AAV
Gao Guang-Ping
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
View Associated: 
Cre recombinase   - Genome Editor
Matched on: Name ->  AAVcc47-Cre vectorName ->  AAVcc47-Cre vectorSubtype ->  AAV experimentName ->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Cre recombinase delivered by AAVcc47-Cre viral vector
View Associated: 
Ai9 mouse (BCM)   - Model System - [In Vivo]
Matched on: Name ->  AAVcc47-CreAAVcc47-SaCas9-Ai9 vectorName ->  AAVcc47-CreAAVcc47-SaCas9-Ai9 vectorSubtype ->  AAV experimentName ->  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.
View Associated: 
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.   - Experiment - [In Vivo]
Matched on: description ->  3e11 vg/mouse of AAV-BI28:GFAP-SaCas9-WPRE-pA and 3e11 vg/mouse of AAV-BI28:GFAP-NLS-GFP-U6-L1-U6-R2 vectorSubtype ->  AAV generatedDescription ->  3e11 vg/mouse of AAV-BI28:GFAP-SaCas9-WPRE-pA and 3e11 vg/mouse of AAV-BI28:GFAP-NLS-GFP-U6-L1-U6-R2 ; sections 4 weeks later. ..............This gRNA targets the Ai9 and related transgenes ..Novel engineered AAV ; fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds..Novel engineered AAV ; ROSA)26Sor locus.................This gRNA targets the Ai9 and related transgenes ..Novel engineered AAV ; fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds..Novel engineered AAV capsidSerotype ->  AAV BI28 (novel engineered variant)
Deverman Benjamin E
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.
View Associated: 
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]
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-Ai9-sgRNA2-CB-SaCas9AAVcc47-Ai9-sgRNA2-CB-SaCas9AAVcc47-Ai9-sgRNA1-CB-SaCas9AAV9-Ai9-sgRNA1-CB-SaCas9 vectorName ->  AAV9-Ai9-sgRNA2-CB-SaCas9AAVcc47-Ai9-sgRNA2-CB-SaCas9AAVcc47-Ai9-sgRNA1-CB-SaCas9AAV9-Ai9-sgRNA1-CB-SaCas9 vectorSubtype ->  AAV generatedDescription ->  and heart. ........................AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus..AAV ; CB SaCas9 targeting the Ai9 locus..AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus..AAV ; 26Sor locus...........................AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus..AAV ; CB SaCas9 targeting the Ai9 locus..AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus..AAV
Asokan Aravind
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.
View Associated: 
SaCas9-Lagor   - Genome Editor
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-Ai9-sgRNA2-CB-SaCas9AAVcc47-Ai9-sgRNA2-CB-SaCas9AAV9-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9AAV9-CMV-SaCas9 vectorName ->  AAV9-Ai9-sgRNA2-CB-SaCas9AAVcc47-Ai9-sgRNA2-CB-SaCas9AAV9-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9AAV9-CMV-SaCas9 vectorSubtype ->  AAV generatedDescription ->  ..............................................................AAV serotype 9 delivering u6 promoter driving ; 1 + sgRNA2 targeting the Ai9 locus..AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus..AAV ; serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus..AAVcc47 delivering CMV driven SaCas9..AAV ; delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene..AAV ; serotype 9 delivering CMV driven SaCas9..AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus..AAV
View Associated: 
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]
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9_pTR_self comp 2xU6-Ai9 guides ; AAV9-CMV-SaCas9AAVcc47_pTR_self comp 2xU6-Ai9 guides vectorName ->  AAV9_pTR_self comp 2xU6-Ai9 guides ; AAV9-CMV-SaCas9AAVcc47_pTR_self comp 2xU6-Ai9 guides vectorSubtype ->  AAV generatedDescription ->  calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. ......................AAV ; delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene..AAV ; fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.........................AAV ; delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene..AAV
Asokan Aravind
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.
View Associated: 
Cre Recombinase dose escalation study in Ai9 mice   - Experiment - [In Vivo]
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-CMV-CreAAVcc47-CMV-Cre vectorName ->  AAV9-CMV-CreAAVcc47-CMV-Cre vectorSubtype ->  AAV generatedDescription ->  ....................................................................................................AAV ; ....................................................................................................AAV
Asokan Aravind
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.
View Associated: 
Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.   - Experiment - [In Vivo]
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-mCherryAAVcc47-mCherry vectorName ->  AAV9-mCherryAAVcc47-mCherry vectorSubtype ->  AAV
Asokan Aravind
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.
View Associated: 
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]
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9AAV9-CMV-SaCas9 vectorName ->  AAV9-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9AAV9-CMV-SaCas9 description ->  single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV vectorSubtype ->  AAV generatedDescription ->  single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV ; calculating percent TdTomato+ cells normalized to Dapi+ cells in liver and heart. ....................AAV ; u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus..AAVcc47 delivering CMV driven SaCas9..AAV ; fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.......................AAV ; u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus..AAVcc47 delivering CMV driven SaCas9..AAV
Asokan Aravind
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.
View Associated: 
Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System   - Study
Matched on: Name ->  Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central
Deverman Benjamin E   Date Of Submission: 2021-04-17
View Associated: 
BI28:AAV-GFAP-SaCas9-WPRE3-pA   - Vector
Matched on: description ->  Novel engineered AAV BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein vectorSubtype ->  AAV generatedDescription ->  ................................This gRNA targets the Ai9 and related transgenes ..Novel engineered AAV ; fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds..Novel engineered AAV capsidSerotype ->  AAV BI28 (novel engineered variant)
Novel engineered AAV BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein (GFAP) promoter
View Associated: 
Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.   - Experiment - [In Vivo]
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAVcc84-GFPAAVcc81-GFPAAV9-GFP vectorName ->  AAVcc84-GFPAAVcc81-GFPAAV9-GFP vectorSubtype ->  AAV
Asokan Aravind
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.
View Associated: 
C57BL/6 mouse (Asokan study)   - Model System - [In Vivo]
Matched on: study ->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Name ->  AAV9-mCherryAAVcc84-GFPAAVcc47-mCherryAAVcc81-GFPAAV9-GFP vectorName ->  AAV9-mCherryAAVcc84-GFPAAVcc47-mCherryAAVcc81-GFPAAV9-GFP vectorSubtype ->  AAV
View Associated: 
BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2   - Vector
Matched on: description ->  Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP vectorSubtype ->  AAV generatedDescription ->  ................................This gRNA targets the Ai9 and related transgenes ..Novel engineered AAV ; fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds..Novel engineered AAV capsidSerotype ->  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
View Associated: 
Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro   - Experiment - [In Vitro]
Matched on: Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 vectorName ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 generatedDescription ->  the mTmG, Ai9 and related transgenes at two sites..This gRNA targets the Ai9 and related transgenes ..AAV ; shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA..AAV shuttle vector with ; the mTmG, Ai9 and related transgenes at two sites..This gRNA targets the Ai9 and related transgenes ..AAV ; shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA..AAV shuttle vector with vectorAnnotatedMap ->  AAV-CMV-SaCas9-U6-modified scaffold-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-R1.gb ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb
Deverman Benjamin E
Reporter transgene activation by SaCas9 gRNA target and modified scaffold sequences by transient transfection in immortalized Ai9 mouse fibroblasts
View Associated: 
Ai9 mouse immortalized fibroblasts   - Model System - [In Vitro]
Matched on: Name ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 vectorName ->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 generatedDescription ->  the mTmG, Ai9 and related transgenes at two sites..This gRNA targets the Ai9 and related transgenes ..AAV ; shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA..AAV shuttle vector with vectorAnnotatedMap ->  AAV-CMV-SaCas9-U6-modified scaffold-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-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
View Associated: 
Deverman_Comprehensive_methods.pdf   - Protocol
Matched on: description ->  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.
Deverman_AAV_production_and_administration_Nat Protocols-Challis_et_al_2019.pdf   - Protocol
Matched on: description ->  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.
AB_1196615    - Antibody - [In Vivo]
Matched on: Name ->  AAV.pU1a-SpCas9 ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  AAV.pU1a-SpCas9 ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorSubtype ->  AAV
GFP (D5.1) XP Rabbit mAb antibody
View Associated: 
AB_2209751    - Antibody - [In Vivo]
Matched on: Name ->  AAV.pU1a-SpCas9 ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  AAV.pU1a-SpCas9 ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorSubtype ->  AAV
Anti-RFP (RABBIT) Antibody
View Associated: 
pAAV.pU1a-SpCas9   - Vector
Matched on: Name ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)
Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS
View Associated: 
H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)   - Vector
Matched on: Name ->  AAV.pU1a-SpCas9 ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  AAV.pU1a-SpCas9 ; H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorSubtype ->  AAV
AAV2/5 expressing SpyCas9. AAV2/5 expressing two sgRNAs under U6 promoter and eGFP
View Associated: 
HEK-293T with Ai9 transient reporter assay   - Model System - [In Vitro]
Matched on: Name ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  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
View Associated: 
Lipofectamine 3000   - Delivery System - [In Vitro]
Matched on: Name ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)
Lipid nanoparticle
View Associated: 
Conklin_Fast-Seq_AAV.pdf   - Protocol
Matched on: description ->  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.
pH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)   - Vector
Matched on: Name ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)
AAV2/5 expressing SpyCas9. AAV2/5 expressing two sgRNAs under U6 promoter and eGFP
View Associated: 
sgAi9L   - Guide - [In Vitro]
Matched on: Name ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)
This sgRNA targets the Ai9 and related transgenes
View Associated: 
sgAi9R   - Guide - [In Vitro]
Matched on: Name ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1) vectorName ->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)
This sgRNA targets the Ai9 and related transgenes
View Associated: 

90 results for aav

Category Type Name Description View Associated..
Guide AAVS1_site_01 Targets AAVS1 safe harbor locus
Guide AAVS1_site_07 Targets AAVS1 safe harbor locus
Guide AAVS1_site_09 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_08 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_10 Targets AAVS1 safe harbor locus
Guide AAVS1_site_11 Targets AAVS1 safe harbor locus
Guide AAVS1_site_02 Targets AAVS1 safe harbor locus
Guide AAVS1_site_03 Targets AAVS1 safe harbor locus
Guide AAVS1_site_13 Targets AAVS1 safe harbor locus
Guide AAVS1_site_12 Targets AAVS1 safe harbor locus
Vector Viral Vector AAV9-CMV-Cre AAV serotype 9 delivering CMV Cre Recombinase
Vector Viral Vector AAV9-GFP AAV2/9 self complementary vector expressing GFP driven by CBh promoter
Vector Viral Vector AAVcc47-Cre AAV2/5 expressing Cre recombinase
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-L3 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 AAV.pU1a-SpCas9 Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS
Vector Viral Vector AAV9-CMV-SaCas9 AAV serotype 9 delivering CMV driven SaCas9
Vector Viral Vector AAVcc47-mCherry AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter
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-L2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
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-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 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 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-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 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-modified scaffold-Sa-L2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
Vector Viral Vector AAV9-Ai9-sgRNA1-CB-SaCas9 AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAV9-Ai9-sgRNA2-CB-SaCas9 AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAVcc47-Ai9-sgRNA1-CB-SaCas9 AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAVcc47-Ai9-sgRNA1 + sgRNA2 AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 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-R2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
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-mCherry AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter
Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
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-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-R1 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
Vector Viral Vector AAVcc84-GFP AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter
Study Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Study Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
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.
Model System Cell CD4/CD8 Human Primary T cell CD4/CD8 Human Primary T cell
Delivery System Commercial Reagent P3 Nucleofection Kit Nuceleofection kit to be used with Lonza's nucleofection system.
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 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.
Genome Editor Cas Nuclease SauCas9
Genome Editor Cas Nuclease SpCas9 HA-SV40NLS-SpCas9-SV40NLS
Genome Editor Cas Nuclease SaCas9
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.
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
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.
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
Genome Editor Recombinase Cre recombinase Cre recombinase delivered by AAVcc47-Cre viral vector
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.
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.
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.
Genome Editor Cas Nuclease SaCas9-Lagor
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.
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.
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.
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.
Study Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System
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
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.
Model System Animal C57BL/6 mouse (Asokan study)
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 Vitro Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Reporter transgene activation by SaCas9 gRNA target and modified scaffold sequences by transient transfection in immortalized Ai9 mouse fibroblasts
Model System Cell Ai9 mouse immortalized fibroblasts Immortalized fibroblasts made from Ai9 (B6.Cg-Gt(ROSA)26Sor^tm9(CAG-tdTomato)Hze/J) mice
Protocol Deverman_Comprehensive_methods.pdf Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue processing and IHC.
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.
Antibody AB_1196615  GFP (D5.1) XP Rabbit mAb antibody
Antibody AB_2209751  Anti-RFP (RABBIT) Antibody
Vector Viral Vector pAAV.pU1a-SpCas9 Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS