Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients
David G. Ousterout(Profectus Biosciences (United States)), Charles A. Gersbach(Duke University), Jacques P. Tremblay(Université Laval), Pratiksha I. Thakore(Broad Institute), Pablo Pérez‐Piñera(University of Illinois Urbana-Champaign), Vincent Mouly(Inserm), Matthew T. Brown(Emory University), Xiaoxia Qin(Duke University), Olivier Fédrigo(Rockefeller University), Ami M. Kabadi(Sarepta Therapeutics (United States))
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