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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-L3 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 vectorName -> 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 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-SaCas9 ; AAVcc47_pTR_self comp 2xU6-Ai9 guides vectorName -> AAV9_pTR_self comp 2xU6-Ai9 guides ; AAV9-Ai9-sgRNA1 + sgRNA2 ; AAV9-CMV-SaCas9 ; AAVcc47_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: Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
AAVcc47-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-L2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 vectorName -> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 ; AAV-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-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 vectorName -> 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 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: Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
AAVcc47-CMV-SaCas9 - Vector 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: Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
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-L1 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2 vectorName -> AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 ; AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 ; AAV-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-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 vectorName -> 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 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-SaCas9 ; AAVcc47_pTR_self comp 2xU6-Ai9 guides vectorName -> AAV9-CMV-SaCas9 ; AAVcc47_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-SaCas9 ; AAV9-Ai9-sgRNA1-CB-SaCas9 vectorName -> AAV9-Ai9-sgRNA2-CB-SaCas9 ; AAV9-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-SaCas9 ; AAV9-Ai9-sgRNA1-CB-SaCas9 vectorName -> AAV9-Ai9-sgRNA2-CB-SaCas9 ; AAV9-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-SaCas9 ; AAVcc47-Ai9-sgRNA1-CB-SaCas9 vectorName -> AAVcc47-Ai9-sgRNA2-CB-SaCas9 ; AAVcc47-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: Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
AAVcc47-CMV-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-L3 ; AAV-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-L3 ; AAV-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-SaCas9 ; AAVcc47-Ai9-sgRNA1-CB-SaCas9 vectorName -> AAVcc47-Ai9-sgRNA2-CB-SaCas9 ; AAVcc47-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: 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) 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.
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: 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.
[] 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-Cre ; AAVcc47-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_14 ; AAVS1_site_13 ; AAVS1_site_12 ; AAVS1_site_11 ; AAVS1_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_14 ; AAVS1_site_13 ; AAVS1_site_12 ; AAVS1_site_11 ; AAVS1_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-Cre ; AAVcc47-CMV-SaCas9 ; AAV9-CMV-SaCas9 ; AAV.pU1a-SpCas9 ; AAVcc47-CMV-Cre vectorName -> AAV9-CMV-Cre ; AAVcc47-CMV-SaCas9 ; AAV9-CMV-SaCas9 ; AAV.pU1a-SpCas9 ; AAVcc47-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: Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System Evolving High Potency AAV Vectors for Neuromuscular Genome Editing BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Testing AAV5 for activation of tdTomato in mouse airway Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Cre Recombinase dose escalation study in Ai9 mice Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) 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 novel human T cell platform to define biological adverse effects of genome editing - Experiment - [In Vitro] Matched on: grnaLabId -> AAVS1_site_14 ; AAVS1_site_13 ; AAVS1_site_12 ; AAVS1_site_11 ; AAVS1_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_14 ; AAVS1_site_13 ; AAVS1_site_12 ; AAVS1_site_11 ; AAVS1_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: Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases A Novel Human T-Cell Platform to Define Biological Effects of Genome Editing Testing AAV5 for activation of tdTomato in HEK293T cells Testing AAV5 for activation of tdTomato in mouse airway A novel human T cell platform to define biological adverse effects of genome editing
SaCas9 - Genome Editor Matched on: Name -> 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 vectorName -> 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 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: Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
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-SaCas9 ; AAV9-CMV-SaCas9 vectorName -> AAV9-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9 ; AAV9-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-Cre ; AAVcc47-SaCas9-Ai9 vectorName -> AAVcc47-Cre ; AAVcc47-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-SaCas9 ; AAVcc47-Ai9-sgRNA2-CB-SaCas9 ; AAVcc47-Ai9-sgRNA1-CB-SaCas9 ; AAV9-Ai9-sgRNA1-CB-SaCas9 vectorName -> AAV9-Ai9-sgRNA2-CB-SaCas9 ; AAVcc47-Ai9-sgRNA2-CB-SaCas9 ; AAVcc47-Ai9-sgRNA1-CB-SaCas9 ; AAV9-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-SaCas9 ; AAVcc47-Ai9-sgRNA2-CB-SaCas9 ; AAV9-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9 ; AAV9-CMV-SaCas9 vectorName -> AAV9-Ai9-sgRNA2-CB-SaCas9 ; AAVcc47-Ai9-sgRNA2-CB-SaCas9 ; AAV9-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9 ; AAV9-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 (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
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-SaCas9 ; AAVcc47_pTR_self comp 2xU6-Ai9 guides vectorName -> AAV9_pTR_self comp 2xU6-Ai9 guides ; AAV9-CMV-SaCas9 ; AAVcc47_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-Cre ; AAVcc47-CMV-Cre vectorName -> AAV9-CMV-Cre ; AAVcc47-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-mCherry ; AAVcc47-mCherry vectorName -> AAV9-mCherry ; AAVcc47-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-SaCas9 ; AAV9-CMV-SaCas9 vectorName -> AAV9-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-Ai9-sgRNA1 + sgRNA2 ; AAVcc47-CMV-SaCas9 ; AAV9-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: Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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: Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
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-GFP ; AAVcc81-GFP ; AAV9-GFP vectorName -> AAVcc84-GFP ; AAVcc81-GFP ; AAV9-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-mCherry ; AAVcc84-GFP ; AAVcc47-mCherry ; AAVcc81-GFP ; AAV9-GFP vectorName -> AAV9-mCherry ; AAVcc84-GFP ; AAVcc47-mCherry ; AAVcc81-GFP ; AAV9-GFP vectorSubtype -> AAV View Associated: 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.
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: Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
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-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 vectorName -> 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 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-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 vectorName -> 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 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: Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Testing AAV5 for activation of tdTomato in mouse airway
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: Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases Expanding CRISPR-Cas Editing Technology through Exploration of Novel Cas Proteins and DNA Repair Systems Testing AAV5 for activation of tdTomato in HEK293T cells Testing newly discovered Biggie Phage editors in human cells
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	Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Vector	Viral Vector	AAVcc47-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	Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Vector	Viral Vector	AAVcc47-CMV-SaCas9	AAVcc47 delivering CMV driven SaCas9	Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Vector	Viral Vector	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	Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)
Vector	Viral Vector	AAVcc47-CMV-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		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) 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.
Study		Evolving High Potency AAV Vectors for Neuromuscular Genome Editing		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.
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.	Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System Evolving High Potency AAV Vectors for Neuromuscular Genome Editing BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Testing AAV5 for activation of tdTomato in mouse airway Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Cre Recombinase dose escalation study in Ai9 mice Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) 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 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	Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases A Novel Human T-Cell Platform to Define Biological Effects of Genome Editing Testing AAV5 for activation of tdTomato in HEK293T cells Testing AAV5 for activation of tdTomato in mouse airway A novel human T cell platform to define biological adverse effects of genome editing
Genome Editor	Cas Nuclease	SaCas9		Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
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		Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector.
Experiment	In Vivo	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		Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
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	Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
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)		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.
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	Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice - Deverman Validation (Deverman, Benjamin) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain
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	Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases The Jackson Laboratory Gene Editing Testing Center - Gong Validation (Gong, Shaoqin) Second site validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain Testing AAV5 for activation of tdTomato in mouse airway
Vector	Viral Vector	pAAV.pU1a-SpCas9	Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS

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90 results for aav

90 results for aav

AAVS1_site_01 - Guide - [In Vitro]

AAVS1_site_07 - Guide - [In Vitro]

AAVS1_site_09 - Guide - [In Vitro]

AAVS1_site_14 - Guide - [In Vitro]

AAVS1_site_04 - Guide - [In Vitro]

AAVS1_site_08 - Guide - [In Vitro]

AAVS1_site_05 - Guide - [In Vitro]

AAVS1_site_06 - Guide - [In Vitro]

AAVS1_site_10 - Guide - [In Vitro]

AAVS1_site_11 - Guide - [In Vitro]

AAVS1_site_02 - Guide - [In Vitro]

AAVS1_site_03 - Guide - [In Vitro]

AAVS1_site_13 - Guide - [In Vitro]

AAVS1_site_12 - Guide - [In Vitro]

AAV9-CMV-Cre - Vector

AAV9-GFP - Vector

AAVcc47-Cre - Vector

AAVcc81-GFP - Vector

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3 - Vector

AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 - Vector

AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2 - Vector

AAV.pU1a-SpCas9 - Vector

AAV9-CMV-SaCas9 - Vector

AAVcc47-mCherry - Vector

AAVcc47-SaCas9-Ai9 - Vector

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2 - Vector

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

AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1 - Vector

AAV9-Ai9-sgRNA1 + sgRNA2 - Vector

AAVcc47-CMV-SaCas9 - Vector

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 - Vector

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1 - Vector

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 - Vector

AAVcc47_pTR_self comp 2xU6-Ai9 guides - Vector

AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 - Vector

AAV9-Ai9-sgRNA1-CB-SaCas9 - Vector

AAV9-Ai9-sgRNA2-CB-SaCas9 - Vector

AAVcc47-Ai9-sgRNA1-CB-SaCas9 - Vector

AAVcc47-Ai9-sgRNA1 + sgRNA2 - Vector

AAVcc47-CMV-Cre - Vector

AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 - Vector

AAV9_pTR_self comp 2xU6-Ai9 guides - Vector

AAV9-mCherry - Vector

AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2 - Vector

AAVcc47-Ai9-sgRNA2-CB-SaCas9 - Vector

AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1 - Vector

AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 - Vector

AAVcc84-GFP - Vector

Evolving High Potency AAV Vectors for Neuromuscular Genome Editing - Study

Evolving High Potency AAV Vectors for Neuromuscular Genome Editing - Study

[] Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. - Experiment - [In Vivo]

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

P3 Nucleofection Kit - Delivery System - [In Vitro]

Ai9 mouse - Model System - [In Vivo]

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

SauCas9 - Genome Editor

SpCas9 - Genome Editor

SaCas9 - Genome Editor

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]

[] Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain - Experiment - [In Vivo]

Testing AAV5 for activation of tdTomato in HEK293T cells - Experiment - [In Vitro]

Testing AAV5 for activation of tdTomato in mouse airway - Experiment - [In Vivo]

Cre recombinase - Genome Editor

Ai9 mouse (BCM) - Model System - [In Vivo]

Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. - 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) - Experiment - [In Vivo]

SaCas9-Lagor - Genome Editor

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]

Cre Recombinase dose escalation study in Ai9 mice - Experiment - [In Vivo]

Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. - 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) - Experiment - [In Vivo]

Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System - Study

BI28:AAV-GFAP-SaCas9-WPRE3-pA - Vector

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

C57BL/6 mouse (Asokan study) - Model System - [In Vivo]

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

Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro - Experiment - [In Vitro]