Reversal of spliceopathy in cardiomyocytes derived from myotonic dystrophy patient-specific iPS cells by gene editing with CRISPR/Cas9 ribonuclear protein complexes

S. Dastidar, D. Majumdar, L. Meert, T. Jans,Y. Fu,D. Reyon,W. Tulalamba,S. Seneca,D. Furling,K. Joung,M. K. Chuah, T. VandenDriessche

HUMAN GENE THERAPY(2018)

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