Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy

Adrian Pickar‐Oliver(Duke University), Veronica Gough(Duke University), Joel D. Bohning(Duke University), Siyan Liu(Duke University), Jacqueline N. Robinson-Hamm(Duke University), Heather Daniels(Duke University), William H. Majoros(Duke University), Garth Devlin(Duke University), Aravind Asokan(Duke University), Charles A. Gersbach(Duke University)
Molecular Therapy
September 10, 2021
10.1016/j.ymthe.2021.09.003
Cited by 65Open Access
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