120 results for aav

AAV   - Delivery System [Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing name -->  AAV  -->  AAV  -->  AAV  -->  AAV9-CMV-Cre  -->  AAV  -->  ....AAV serotype 9 delivering CMV Cre Recombinase..See vector details..CAG-loxP-stop-loxP-tdtomato reporter name -->  AAV
See vector details
AAVS1_site_05   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_05  -->  AAVS1_site_05 name -->  AAVS1_site_05  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_06   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_06  -->  AAVS1_site_06 name -->  AAVS1_site_06  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_14   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_14  -->  AAVS1_site_14 name -->  AAVS1_site_14  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_01   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_01  -->  AAVS1_site_01 name -->  AAVS1_site_01  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_03   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_03  -->  AAVS1_site_03 name -->  AAVS1_site_03  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_04   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_04  -->  AAVS1_site_04 name -->  AAVS1_site_04  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_11   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_11  -->  AAVS1_site_11 name -->  AAVS1_site_11  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_12   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_12  -->  AAVS1_site_12 name -->  AAVS1_site_12  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_08   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_08  -->  AAVS1_site_08 name -->  AAVS1_site_08  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_10   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_10  -->  AAVS1_site_10 name -->  AAVS1_site_10  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_09   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_09  -->  AAVS1_site_09 name -->  AAVS1_site_09  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_13   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_13  -->  AAVS1_site_13 name -->  AAVS1_site_13  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_02   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_02  -->  AAVS1_site_02 name -->  AAVS1_site_02  -->  AAVS1
Targets AAVS1 safe harbor locus
AAVS1_site_07   - Guide [Biological Systems] - [In Vitro]
Matched on: name -->  AAVS1_site_07  -->  AAVS1_site_07 name -->  AAVS1_site_07  -->  AAVS1
Targets AAVS1 safe harbor locus
AAV9-CMV-SaCas9   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAV9-CMV-SaCas9  -->  AAV9_pTR_self comp 2xU6-Ai9 guides  AAV9-Ai9-sgRNA1 + sgRNA2  AAV9-CMV-SaCas9  AAVcc47_pTR_self comp 2xU6-Ai9 guides  -->  AAV serotype 9 delivering CMV driven SaCas9  -->  AAV  -->  ..................................AAV serotype 9 delivering 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
AAVcc47-Ai9-sgRNA1 + sgRNA2   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc47-Ai9-sgRNA1 + sgRNA2  -->  AAVcc47-Ai9-sgRNA1 + sgRNA2  AAVcc47-CMV-SaCas9  -->  AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus  -->  AAV  -->  ..............AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus.
AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus
AAVcc47-Ai9-sgRNA2-CB-SaCas9   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc47-Ai9-sgRNA2-CB-SaCas9  -->  AAVcc47-Ai9-sgRNA2-CB-SaCas9  AAVcc47-Ai9-sgRNA1-CB-SaCas9  -->  AAV
AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA  -->  ..This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA  -->  ..This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA  -->  ........This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold  -->  ......This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold  -->  ......This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold  -->  ........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  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
AAV9-CMV-Cre   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV symbol -->  AAV9-CMV-Cre  -->  AAV9-CMV-Cre  -->  AAV serotype 9 delivering CMV Cre Recombinase  -->  AAV  -->  ........................................AAV serotype 9 delivering CMV Cre Recombinase..See vector details
AAV serotype 9 delivering CMV Cre Recombinase
AAV9_pTR_self comp 2xU6-Ai9 guides   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAV9_pTR_self comp 2xU6-Ai9 guides  -->  AAV9_pTR_self comp 2xU6-Ai9 guides  AAV9-CMV-SaCas9  -->  AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting  -->  AAV  -->  ..........AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector)  targeting Ai9 transgene..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
AAVcc47-Ai9-sgRNA1-CB-SaCas9   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc47-Ai9-sgRNA1-CB-SaCas9  -->  AAVcc47-Ai9-sgRNA2-CB-SaCas9  AAVcc47-Ai9-sgRNA1-CB-SaCas9  -->  AAV
AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA  -->  ........This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold  -->  ..This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
AAV.pU1a-SpCas9   - Vector 
Matched on: symbol -->  AAV.pU1a-SpCas9  -->  AAV.pU1a-SpCas9  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV
Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS
AAV9-Ai9-sgRNA2-CB-SaCas9   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAV9-Ai9-sgRNA2-CB-SaCas9  -->  AAV9-Ai9-sgRNA2-CB-SaCas9  AAV9-Ai9-sgRNA1-CB-SaCas9  -->  AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus  -->  AAV  -->  ..........AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus..AAV serotype 9 delivering
AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus
AAVcc47-CMV-Cre   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc47-CMV-Cre  -->  AAVcc47-CMV-Cre  -->  AAV
AAVcc47 delivering CMV Cre Recombinase
AAVcc47-CMV-SaCas9   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc47-CMV-SaCas9  -->  AAVcc47-Ai9-sgRNA1 + sgRNA2  AAVcc47-CMV-SaCas9  -->  AAV  -->  ..............AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus.
AAVcc47 delivering CMV driven SaCas9
AAVcc47-SaCas9-Ai9   - Vector  [Small Animal Testing Center (SATC)]
Matched on: symbol -->  AAVcc47-SaCas9-Ai9  -->  AAVcc47-SaCas9-Ai9  -->  AAV  -->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
AAV2/9 expressing SaCas9 and single sgRNA under U6 promoter
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA  -->  ......This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA  -->  ........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  -->  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
AAV9-Ai9-sgRNA1 + sgRNA2   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAV9-Ai9-sgRNA1 + sgRNA2  -->  AAV9-Ai9-sgRNA1 + sgRNA2  AAV9-CMV-SaCas9  -->  AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus  -->  AAV  -->  ..................AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 targeting the Ai9 locus  ..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
AAVcc47_pTR_self comp 2xU6-Ai9 guides   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc47_pTR_self comp 2xU6-Ai9 guides  -->  AAV9-CMV-SaCas9  AAVcc47_pTR_self comp 2xU6-Ai9 guides  -->  AAV  -->  ..........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
AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA  -->  ......This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold  -->  ..This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter  -->  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
AAV9-mCherry   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAV9-mCherry  -->  AAV9-mCherry  -->  AAV
AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter
AAVcc47-Cre   - Vector  [Small Animal Testing Center (SATC)]
Matched on: symbol -->  AAVcc47-Cre  -->  AAVcc47-Cre  -->  AAV  -->  Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details  )..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector  -->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
AAV2/5 expressing Cre recombinase
AAVcc47-mCherry   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc47-mCherry  -->  AAVcc47-mCherry  -->  AAV
AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter
AAVcc84-GFP   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc84-GFP  -->  AAVcc84-GFP  -->  AAV
AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter
AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold  -->  ........This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven  -->  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
AAV9-Ai9-sgRNA1-CB-SaCas9   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAV9-Ai9-sgRNA1-CB-SaCas9  -->  AAV9-Ai9-sgRNA2-CB-SaCas9  AAV9-Ai9-sgRNA1-CB-SaCas9  -->  AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus  -->  AAV  -->  ..........AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus..AAV serotype 9 delivering
AAV serotype 9 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
AAVcc81-GFP   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAVcc81-GFP  -->  AAVcc81-GFP  -->  AAV
AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter
AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3   - Vector  [Delivery Systems Initiative]
Matched on: symbol -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3  -->  AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold  -->  ....This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven SaCas9  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3.gb
AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
AAV9-GFP   - Vector  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing symbol -->  AAV9-GFP  -->  AAV9-GFP  -->  AAV
AAV2/9 self complementary vector expressing GFP driven by CBh promoter
Evolving High Potency AAV Vectors for Neuromuscular Genome Editing   - Project [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  Recombinant adeno-associated viruses (AAV) have emerged as safe and effective vectors for clinical gene  The current proposal is on a comprehensive and innovative approach to evolve high potency AAV variants name -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Asokan Aravind NIH Report
Recombinant adeno-associated viruses (AAV) have emerged as safe and effective vectors for clinical gene therapy applications including systemic treatment of neuromuscular diseases such as Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD), and Giant Axonal Neuropathy (GAN) amongst others. However, genome editing in neuromuscular tissue, in particular, is challenging. The current proposal is on a comprehensive and innovative approach to evolve high potency AAV variants for systemic neuromuscular genome editing.
[Validation] Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.   - Experiment [Small Animal Testing Center (SATC)] - [In Vivo]
Matched on:  -->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAVcc47-Cre  AAVcc47-SaCas9-Ai9  -->  Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery.  -->  AAV  -->  )..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector  details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see  AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered  by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase name -->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Heaney Jason D
Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues.
Ai9 mouse   - Model System [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV  -->  AAV9-CMV-Cre  AAVcc47-CMV-SaCas9  AAV9-CMV-SaCas9  AAV.pU1a-SpCas9  AAVcc47-CMV-Cre  -->  AAV  -->  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  AAV2/5 expressing two sgRNAs under U6 promoter and eGFP..Novel engineered AAV BI28 variant expressing  delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene..AAV  -->  AAV BI28 (novel engineered variant)
Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.
Cre Recombinase dose escalation study in Ai9 mice   - Experiment [Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV  -->  AAV9-CMV-Cre  AAVcc47-CMV-Cre  -->  AAV  -->  ....................................................................................................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.
A novel human T cell platform to define biological adverse effects of genome editing   - Experiment [Biological Systems] - [In Vitro]
Matched on:  -->  AAVS1_site_14  AAVS1_site_13  AAVS1_site_12  AAVS1_site_11  AAVS1_site_10  -->  AAVS1
Tsai Shengdar Q
110 guide RNAs and SpCas9 were transfected into human T-cells. Indel rates were measured by targeted amplicon deep sequencing.
P3 Nucleofection Kit   - Delivery System [Biological Systems] - [In Vitro]
Matched on:  -->  AAVS1_site_14  AAVS1_site_13  AAVS1_site_12  AAVS1_site_11  AAVS1_site_10  -->  AAVS1
Nuceleofection kit to be used with Lonza's nucleofection system.
CD4/CD8 Human Primary T cell   - Model System [Biological Systems] - [In Vitro]
Matched on:  -->  AAVS1_site_14  AAVS1_site_13  AAVS1_site_12  AAVS1_site_11  AAVS1_site_10  -->  AAVS1
CD4/CD8 Human Primary T cell
SpCas9   - Genome Editor  [Biological Systems]
Matched on:  -->  AAVS1_site_14  AAVS1_site_13  AAVS1_site_12  AAVS1_site_11  AAVS1_site_10  -->  AAV.pU1a-SpCas9  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV  -->  AAVS1
HA-SV40NLS-SpCas9-SV40NLS
[Validation] Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice   - Experiment [Small Animal Testing Center (SATC)] - [In Vivo]
Matched on:  -->  AAV  -->  by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase
Heaney Jason D
AAV5 encoding CRISPR/Cas editing machinery were delivered to the lungs of reporter mice by intratracheal instillation. After 4 weeks incubation, the mice were dissected and the lungs imaged for the presence of tdTomato fluorescence, indicating successful editing. Editing calculated by dividing the number of tdTomato+ red cells by the number of nuclei in each airway
scAAV5-Cre-GFP   - Vector  [Small Animal Testing Center (SATC)]
Matched on:  -->  AAV  -->  Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details  )..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector  details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter)   - Experiment [Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV9-Ai9-sgRNA1 + sgRNA2  AAVcc47-Ai9-sgRNA1 + sgRNA2  AAVcc47-CMV-SaCas9  AAV9-CMV-SaCas9  -->  single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV  -->  AAV  -->  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 name -->  Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to
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.
Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)   - Experiment [Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV9-Ai9-sgRNA1 + sgRNA2  AAVcc47-Ai9-sgRNA1 + sgRNA2  AAVcc47-CMV-SaCas9  AAV9-CMV-SaCas9  -->  single guide RNAs targeting the Rosa26 locus and one delivering CMV driven SaCas9 (both single stranded AAV  -->  AAV  -->  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 name -->  Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9
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.
SauCas9   - Genome Editor  [Small Animal Testing Center (SATC)]
Matched on:  -->  AAVcc47-SaCas9-Ai9  -->  AAV  -->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Ai9 SaCas9 Guide B   - Guide [Small Animal Testing Center (SATC)] - [In Vivo]
Matched on:  -->  AAVcc47-SaCas9-Ai9  -->  AAV  -->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
This gRNA targets the Ai9 and related transgenes
Ai9 SaCas9 Guide A   - Guide [Small Animal Testing Center (SATC)] - [In Vivo]
Matched on:  -->  AAVcc47-SaCas9-Ai9  -->  AAV  -->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
This gRNA targets the Ai9 and related transgenes
Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro   - Experiment [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  -->  by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb
Deverman Benjamin E
Reporter transgene activation by SaCas9 gRNA target and modified scaffold sequences by transient transfection in immortalized Ai9 mouse fibroblasts
Ai9 mouse (BCM)   - Model System [Small Animal Testing Center (SATC)] - [In Vivo]
Matched on:  -->  AAVcc47-Cre  AAVcc47-SaCas9-Ai9  -->  AAV  -->  Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details  )..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector  details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see  AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered  by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  -->  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.
Ai9-SauSpyCas9 mouse   - Model System [Small Animal Testing Center (SATC)] - [In Vivo]
Matched on:  -->  AAV  -->  Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details  )..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector  details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see  AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered  by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase
Ai9-SauSpyCas9 is a version of Ai9 that has a single guide RNA sites on both sides of the STOP cassette. This is a Cre reporter allele that has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Ai9 mice express robust tdTomato fluorescence following Cre-mediated recombination.
R2-modified   - Guide [Delivery Systems Initiative] - [In Vivo, In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1  -->  AAV  -->  This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven SaCas9  and U6 promoter driven gRNA..Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial  fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds..Novel engineered AAV  BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein (GFAP) promoter ..AAV  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb  -->  AAV BI28 (novel engineered variant)
This gRNA targets the Ai9 and related transgenes
Ai9 mouse immortalized fibroblasts   - Model System [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  -->  by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase  delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..This gRNA  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb
Immortalized fibroblasts made from Ai9 (B6.Cg-Gt(ROSA)26Sor^tm9(CAG-tdTomato)Hze/J) mice
[Validation] Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain   - Experiment [Small Animal Testing Center (SATC)] - [In Vivo]
Matched on:  -->  Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing  -->  AAV  -->  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  -->  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
SaCas9-Lagor   - Genome Editor  [Delivery Systems Initiative]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV9-Ai9-sgRNA2-CB-SaCas9  AAV9-Ai9-sgRNA1 + sgRNA2  AAVcc47-CMV-SaCas9  AAV9-Ai9-sgRNA1-CB-SaCas9  AAV9-CMV-SaCas9  -->  AAV  -->  ..............................................................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
Cre recombinase   - Genome Editor  [Small Animal Testing Center (SATC), Delivery Systems Initiative]
Matched on:  -->  AAVcc47-Cre  -->  Cre recombinase delivered by AAV (see vector details)  -->  AAV  -->  Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details  )..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see vector  details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered by AAV (see  AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)..Cre recombinase delivered  by AAV (see vector details)..Cre recombinase delivered by AAV (see vector details)....AAV2/5 expressing  -->  Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
Cre recombinase delivered by AAV (see vector details)
L2-modified   - Guide [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo, In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2  -->  AAV  -->  This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven SaCas9  and U6 promoter driven gRNA with modified scaffold sequence..Novel engineered AAV BI28 variant expressing  fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds..Novel engineered AAV  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb  -->  AAV BI28 (novel engineered variant)
This gRNA targets the Ai9 and related transgenes
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 [Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV9_pTR_self comp 2xU6-Ai9 guides  AAV9-CMV-SaCas9  AAVcc47_pTR_self comp 2xU6-Ai9 guides  -->  AAV  -->  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 name -->  Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to
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.
SaCas9   - Genome Editor  [Small Animal Testing Center (SATC), Delivery Systems Initiative]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  -->  AAV  -->  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  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  -->  AAV BI28 (novel engineered variant)
Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter)   - Experiment [Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV9-Ai9-sgRNA2-CB-SaCas9  AAVcc47-Ai9-sgRNA2-CB-SaCas9  AAVcc47-Ai9-sgRNA1-CB-SaCas9  AAV9-Ai9-sgRNA1-CB-SaCas9  -->  AAV  -->  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 name -->  Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to
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.
BI28:AAV-GFAP-SaCas9-WPRE3-pA   - Vector  [Small Animal Testing Center (SATC), Delivery Systems Initiative]
Matched on:  -->  Novel engineered AAV BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein  -->  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  -->  AAV BI28 (novel engineered variant)
Novel engineered AAV BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein (GFAP) promoter
Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.   - Experiment [Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAVcc84-GFP  AAVcc81-GFP  AAV9-GFP  -->  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.
C57BL/6 mouse (Asokan study)   - Model System [Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV9-mCherry  AAVcc47-mCherry  AAVcc84-GFP  AAVcc81-GFP  AAV9-GFP  -->  AAV
Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.   - Experiment [Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  Evolving High Potency AAV Vectors for Neuromuscular Genome Editing  -->  AAV9-mCherry  AAVcc47-mCherry  -->  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.
Heaney-SATC_Gao-Validation_Intratracheal_Delivery_of_AAV_in_Mice.pdf   - Protocol 
Matched on:  -->  Procedure for Intratracheal (IT) delivery of AAV in mouse lung.
Procedure for Intratracheal (IT) delivery of AAV in mouse lung.
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.   - Experiment [Delivery Systems Initiative] - [In Vivo]
Matched on:  -->  3e11 vg/mouse of AAV-BI28:GFAP-SaCas9-WPRE-pA and 3e11 vg/mouse of AAV-BI28:GFAP-NLS-GFP-U6-L1-U6-R2  -->  AAV  -->  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  -->  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.
L3-unmodified   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven SaCas9  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb
This gRNA targets the Ai9 and related transgenes
R1-modified   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1  -->  This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven SaCas9  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb
This gRNA targets the Ai9 and related transgenes
SaLoxP1-modified   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1  -->  This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1.gb
This gRNA targets the mTmG, Ai9 and related transgenes at two sites
SaLoxP2-modified   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2  -->  This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2.gb
This gRNA targets the mTmG, Ai9 and related transgenes at two sites
L3-modifed   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3  -->  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 driven  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3.gb
This gRNA targets the Ai9 and related transgenes
L1-modified   - Guide [Small Animal Testing Center (SATC), Delivery Systems Initiative] - [In Vivo, In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1  -->  AAV  -->  This gRNA targets the Ai9 and related transgenes ..Novel engineered AAV BI28 variant expressing NLS-GFP  fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds..Novel engineered AAV  BI28 variant expressing S. aureus Cas9 driven by the glial fibrillary acidic protein (GFAP) promoter ..AAV  -->  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2.gb  AAV-CMV-SaCas9-U6-modified scaffold-Sa-L1.gb  -->  AAV BI28 (novel engineered variant)
This gRNA targets the Ai9 and related transgenes
Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System   - Project [Delivery Systems Initiative]
Matched on: name -->  Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central
Deverman Benjamin E NIH Report
This project aims to advance the NIH Somatic Cell Genome Editing Program’s objective to identify novel delivery technologies that enable genome editing in therapeutically relevant somatic cell populations. We will use proven virus engineering methods to develop new vehicles that can deliver genome editing machinery throughout the adult mammalian central nervous system. Accomplishing this objective would pave the road for applying gene editing, and gene therapy more broadly, to the study and treatment of neurological and psychiatric disorders.
Deverman_AAV_production_and_administration_Nat Protocols-Challis_et_al_2019.pdf   - Protocol 
Matched on:  -->  Published procedures for AAV production, delivery, and tissue imaging. Challis et al.  Systemic AAV vectors for widespread and targeted gene delivery in rodents.
Published procedures for AAV production, delivery, and tissue imaging. Challis et al. Systemic AAV vectors for widespread and targeted gene delivery in rodents. Nat Protoc. 2019 Feb;14(2):379-414. doi: 10.1038/s41596-018-0097-3.
BI28:AAV-GFAP-NLS-GFP-WPRE-synpA-L1-R2   - Vector  [Small Animal Testing Center (SATC), Delivery Systems Initiative]
Matched on:  -->  Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP  -->  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  -->  AAV BI28 (novel engineered variant)
Novel engineered AAV BI28 variant expressing NLS-GFP driven by the glial fibrillary acidic protein (GFAP) promoter and dual sgRNAs with modified scaffolds
R2-unmodified   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven SaCas9  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb
This gRNA targets the Ai9 and related transgenes
SaLoxP1-unmodified   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1  -->  This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP1.gb
This gRNA targets the mTmG, Ai9 and related transgenes at two sites
L1-unmodified   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven SaCas9  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb
This gRNA targets the Ai9 and related transgenes
L2-unmodified   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2  -->  This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven SaCas9  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb
This gRNA targets the Ai9 and related transgenes
R1-unmodified   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1  -->  This gRNA targets the Ai9 and related transgenes ..AAV shuttle vector with CMV promoter driven SaCas9  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L2.gb  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L3.gb
This gRNA targets the Ai9 and related transgenes
SaLoxP2-unmodified   - Guide [Delivery Systems Initiative] - [In Vitro]
Matched on:  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2  -->  This gRNA targets the mTmG, Ai9 and related transgenes at two sites..AAV shuttle vector with CMV promoter  -->  AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-LoxP2.gb
This gRNA targets the mTmG, Ai9 and related transgenes at two sites
Deverman_Comprehensive_methods.pdf   - Protocol 
Matched on:  -->  Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue
Procedure for plasmid cloning, editing evaluation in fibroblast, AAV production and administration, tissue processing and IHC.
Conklin_Fast-Seq_AAV.pdf   - Protocol 
Matched on:  -->  Published procedure for AAV composition measurement using fast-seq. Maynard et al.
Published procedure for AAV composition measurement using fast-seq. Maynard et al. Fast-Seq: A Simple Method for Rapid and Inexpensive Validation of Packaged Single-Stranded Adeno-Associated Viral Genomes in Academic Settings. Hum Gene Ther Methods . 2019 Dec;30(6):195-205. doi: 10.1089/hgtb.2019.110.
H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)   - Vector 
Matched on:  -->  AAV.pU1a-SpCas9  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV
AAV2/5 expressing SpyCas9. AAV2/5 expressing two sgRNAs under U6 promoter and eGFP
Matched on:  -->  AAV.pU1a-SpCas9  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV
Other Id:   Cell Signaling Technology Cat# 2956 
GFP (D5.1) XP Rabbit mAb antibody
Matched on:  -->  AAV.pU1a-SpCas9  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV
Other Id:   Rockland Cat# 600-401-379 
Anti-RFP (RABBIT) Antibody
Matched on:  -->  AAV.pU1a-SpCas9  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV
Other Id:   Santa Cruz, SC-25555 
Anti-CC10 (Rabbit) Polyclonal Antibody, dilution used 1:2,000
pAAV.pU1a-SpCas9   - Vector 
Matched on:  -->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)
Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS
sgAi9L   - Guide 
Matched on:  -->  AAV.pU1a-SpCas9  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV
This sgRNA targets the Ai9 and related transgenes
sgAi9R   - Guide 
Matched on:  -->  AAV.pU1a-SpCas9  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV
This sgRNA targets the Ai9 and related transgenes
Matched on:  -->  AAV.pU1a-SpCas9  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV
Other Id:   Thermo Fisher Scientific, MA5-15257 
Anti-RFP (Mouse) Monoclonal Antibody, dilution used 1:300
Matched on:  -->  AAV.pU1a-SpCas9  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV
Other Id:   Rockland, 600- 401-379 
Anti-RFP (Rabbit) Polyclonal Antibody
Matched on:  -->  AAV.pU1a-SpCas9  H509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)  -->  AAV
Other Id:   Santa Cruz, SC-23950 
Anti-alpha Tubulin (Mouse) Monoclonal Antibody, dilution used 1:200
HEK-293T with Ai9 transient reporter assay   - Model System 
Matched on:  -->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)
HEK-293T cells transfected with an Ai9 inducible transgene reporter plasmid used to test gene editing activity by fluorescence
SaCas9   - Genome Editor  [Small Animal Testing Center (SATC)]
Matched on:  -->  AAV
BCM_ssAAV5-Sp_sgA   - Guide [Small Animal Testing Center (SATC)] - [In Vivo]
Matched on:  -->  AAV
This sgRNA targets the Ai9 and related transgenes
Lipofectamine 3000   - Delivery System [Genome Editors] - [In Vitro]
Matched on:  -->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)
Lipid nanoparticle
pH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)   - Vector 
Matched on:  -->  PH509 AAVsc-u6-sgAI9L-U6-AI9R-U1A-EGFP (1)
AAV2/5 expressing SpyCas9. AAV2/5 expressing two sgRNAs under U6 promoter and eGFP
BCM_ssAAV5-Sa_sgB   - Guide [Small Animal Testing Center (SATC)] - [In Vivo]
Matched on:  -->  AAV
This gRNA targets the Ai9 and related transgenes
BCM_ssAAV5-Sp_sgB   - Guide [Small Animal Testing Center (SATC)] - [In Vivo]
Matched on:  -->  AAV
This sgRNA targets the Ai9 and related transgenes

120 results for aav

Category Type Name Description View Associated..
Delivery System Viral vector AAV See vector details
Guide AAVS1_site_05 Targets AAVS1 safe harbor locus
Guide AAVS1_site_06 Targets AAVS1 safe harbor locus
Guide AAVS1_site_14 Targets AAVS1 safe harbor locus
Guide AAVS1_site_01 Targets AAVS1 safe harbor locus
Guide AAVS1_site_03 Targets AAVS1 safe harbor locus
Guide AAVS1_site_04 Targets AAVS1 safe harbor locus
Guide AAVS1_site_11 Targets AAVS1 safe harbor locus
Guide AAVS1_site_12 Targets AAVS1 safe harbor locus
Guide AAVS1_site_08 Targets AAVS1 safe harbor locus
Guide AAVS1_site_10 Targets AAVS1 safe harbor locus
Guide AAVS1_site_09 Targets AAVS1 safe harbor locus
Guide AAVS1_site_13 Targets AAVS1 safe harbor locus
Guide AAVS1_site_02 Targets AAVS1 safe harbor locus
Guide AAVS1_site_07 Targets AAVS1 safe harbor locus
Vector Viral Vector AAV9-CMV-SaCas9 AAV serotype 9 delivering CMV driven SaCas9
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-Ai9-sgRNA2-CB-SaCas9 AAVcc47 delivering sgRNA 2 + CB SaCas9 targeting the Ai9 locus
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-LoxP2 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 AAV-CMV-SaCas9-U6-modified scaffold-Sa-L2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
Vector Viral Vector AAV-CMV-SaCas9-U6-modified scaffold-Sa-R2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
Vector Viral Vector AAV9-CMV-Cre AAV serotype 9 delivering CMV Cre Recombinase
Vector Viral Vector AAV9_pTR_self comp 2xU6-Ai9 guides AAV serotype 9 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene
Vector Viral Vector AAVcc47-Ai9-sgRNA1-CB-SaCas9 AAVcc47 delivering sgRNA 1 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-R1 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
Vector Viral Vector AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP1 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
Vector Viral Vector AAV.pU1a-SpCas9 Expresses codon-optimized SpCas9 in mammalian cells. HA-SV40NLS-SpCas9-SV40NLS
Vector Viral Vector AAV9-Ai9-sgRNA2-CB-SaCas9 AAV serotype 9 delivering gRNA 2 + CB SaCas9 targeting the Ai9 locus
Vector Viral Vector AAVcc47-CMV-Cre AAVcc47 delivering CMV Cre Recombinase
Vector Viral Vector AAVcc47-CMV-SaCas9 AAVcc47 delivering CMV driven SaCas9
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-L3 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
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_pTR_self comp 2xU6-Ai9 guides AAVcc47 delivering u6 promoter driving sgRNA 1 + sgRNA2 (self complementray vector) targeting Ai9 transgene
Vector Viral Vector AAV-CMV-SaCas9-U6-gRNA-LoxP-Sa-L1 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA
Vector Viral Vector AAV-CMV-SaCas9-U6-modified scaffold-Sa-LoxP2 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
Vector Viral Vector AAV9-mCherry AAV2/9 self complementary vector expressing Mcherry driven by CBh promoter
Vector Viral Vector AAVcc47-Cre AAV2/5 expressing Cre recombinase
Vector Viral Vector AAVcc47-mCherry AAV2/9 self complementary vector with capsid variant cc47 expressing Mcherry driven by CBh promoter
Vector Viral Vector AAVcc84-GFP AAV2/9 self complementary vector with capsid variant cc84 expressing GFP driven by CBh promoter
Vector Viral Vector AAV-CMV-SaCas9-U6-modified scaffold-Sa-R1 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 AAVcc81-GFP AAV2/9 self complementary vector with capsid variant cc81 expressing GFP driven by CBh promoter
Vector Viral Vector AAV-CMV-SaCas9-U6-modified scaffold-Sa-L3 AAV shuttle vector with CMV promoter driven SaCas9 and U6 promoter driven gRNA with modified scaffold sequence
Vector Viral Vector AAV9-GFP AAV2/9 self complementary vector expressing GFP driven by CBh promoter
Project Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Recombinant adeno-associated viruses (AAV) have emerged as safe and effective vectors for clinical gene therapy applications including systemic treatment of neuromuscular diseases such as Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD), and Giant Axonal Neuropathy (GAN) amongst others. However, genome editing in neuromuscular tissue, in particular, is challenging. The current proposal is on a comprehensive and innovative approach to evolve high potency AAV variants for systemic neuromuscular genome editing.
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 Animal Ai9 mouse Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.
Experiment In Vivo 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 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.
Delivery System Commercial Reagent P3 Nucleofection Kit Nuceleofection kit to be used with Lonza's nucleofection system.
Model System Cell CD4/CD8 Human Primary T cell CD4/CD8 Human Primary T cell
Genome Editor Cas Nuclease SpCas9 HA-SV40NLS-SpCas9-SV40NLS
Experiment In Vivo Validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice AAV5 encoding CRISPR/Cas editing machinery were delivered to the lungs of reporter mice by intratracheal instillation. After 4 weeks incubation, the mice were dissected and the lungs imaged for the presence of tdTomato fluorescence, indicating successful editing. Editing calculated by dividing the number of tdTomato+ red cells by the number of nuclei in each airway
Vector Viral Vector scAAV5-Cre-GFP
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.
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.
Genome Editor Cas Nuclease SauCas9
Guide Ai9 SaCas9 Guide B This gRNA targets the Ai9 and related transgenes
Guide Ai9 SaCas9 Guide A This gRNA targets the Ai9 and related transgenes
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 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.
Model System Animal Ai9-SauSpyCas9 mouse Ai9-SauSpyCas9 is a version of Ai9 that has a single guide RNA sites on both sides of the STOP cassette. This is a Cre reporter allele that has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Ai9 mice express robust tdTomato fluorescence following Cre-mediated recombination.
Guide R2-modified This gRNA targets the Ai9 and related transgenes
Model System Cell Ai9 mouse immortalized fibroblasts Immortalized fibroblasts made from Ai9 (B6.Cg-Gt(ROSA)26Sor^tm9(CAG-tdTomato)Hze/J) mice
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
Genome Editor Cas Nuclease SaCas9-Lagor
Genome Editor Recombinase Cre recombinase Cre recombinase delivered by AAV (see vector details)
Guide L2-modified This gRNA targets the Ai9 and related transgenes