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In vivo gene editing in dystrophic mouse muscle and muscle stem cells.
Tabebordbar M, Zhu K, Cheng JK, Chew WL, Widrick JJ, Yan WX, Maesner C, Wu EY, Xiao R, Ran FA, Cong L, Zhang F, Vandenberghe LH, Church GM, Wagers AJ
Science. 2016 Jan 22;351(6271):407-11. doi: 10.1126/science.aad5177. Epub 2015 Dec 31.
PubMed Article

Plasmids from Article

ID Plasmid Purpose  
78601pZac2.1 SV40-CMV-SaCas9-3xNLSAAV vector containing SaCas9
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78603pZac2.1 U6-SaDMDR7-U6-SaDMDL2AAV vector containing gRNAs (for SaCas9) targeting Dmd introns 22 and 23
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78604pZac2.1 U6-SaAi9L-U6-SaAi9RAAV vector containing gRNAs (for SaCas9) targeting Ai9 stop cassette
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78606pZac2.1 CMV173-SaCas9-U6-SaDMDR7-U6-SaDMDL2pZac2.1 CMV173-SaCas9-U6-SaDMDR7-U6-SaDMDL2
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78607pZac2.1 EFs-SaCas9-U6-Sa DMDR7-U6-SaDMDL2A single vector AAV-Cas9 system containing SaCas9 under EFs promoter, gRNAs targeting Dmd introns 22 and 23
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78608pCRII-Topo U6-SpAi9L-U6SpDMDLExpresses gRNAs (for SpCas9) targeting 5’ of Ai9 stop cassette and Dmd intron 22
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78609pCRII-Topo U6-SpAi9R-U6-SpDMDRExpresses gRNAs (for SpCas9) targeting 3’ of Ai9 stop cassette and Dmd intron 23
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