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Improved bi-allelic modification of a transcriptionally silent locus in patient-derived iPSC by Cas9 nickase.

Eggenschwiler R, Moslem M, Fraguas MS, Galla M, Papp O, Naujock M, Fonfara I, Gensch I, Wahner A, Beh-Pajooh A, Mussolino C, Tauscher M, Steinemann D, Wegner F, Petri S, Schambach A, Charpentier E, Cathomen T, Cantz T
Sci Rep. 2016 Dec 2;6:38198. doi: 10.1038/srep38198. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
86003AAT-PB-CG2APtkhomology donor for AAT locus with piggyBac-flanked eGFP_T2A_puroTK cassette
86004AAT-PB-CD2APtkhomology donor for AAT locus with piggyBac-flanked DsRedEx_T2A_puroTK cassette
86005AAT_g1 gRNAplasmid vector encoding for U6-driven AAT_g1 gRNA
86006AAT_g2 gRNAplasmid vector encoding for U6-driven AAT_g2 gRNA (Z-allele specific)
86007Lenti CGIP + Z-AAT targetlenti reporter plasmid with Z-AAT-specific gRNA target-sequence, to be used with tGFP #26864
86008Lenti CGIP + M-AAT targetlenti reporter plasmid with M-AAT-specific gRNA target-sequence, to be used with tGFP #26864
86009Lenti CGIP + TTR targetlenti reporter plasmid with TTR_V30M-specific gRNA target-sequence, to be used with tGFP #26864

Antibodies from Article