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Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9.

Li HL, Fujimoto N, Sasakawa N, Shirai S, Ohkame T, Sakuma T, Tanaka M, Amano N, Watanabe A, Sakurai H, Yamamoto T, Yamanaka S, Hotta A
Stem Cell Reports. 2014 Nov 25. pii: S2213-6711(14)00335-X. doi: 10.1016/j.stemcr.2014.10.013. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
60599pHL-EF1a-SphcCas9-iP-AExpresses human codon-optimized Cas9 (derived from Streptococcus pyogenes) and pruomycin resistance gene.
60600pHL-EF1a-SphcCas9(D10A)-iP-AExpresses D10A mutant (nickase) of human codon-optimized Cas9 (derived from Streptococcus pyogenes) and pruomycin resistance gene.
60601pHL-H1-ccdB-mEF1a-RiHCloning vector for CRISPR-sgRNA (into the BamHI-EcoRI site), expresses RFP and hygromycin resistance gene.
60605pENTR-DMD-DonorKnock-in donor vector to insert Exon 44 in front of Exon 45 of human DYSTROPHIN (DMD)gene, include EF1a-hygromycin resistance cassette flanked by loxP sequences.

Antibodies from Article