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

por Pickar-Oliver, Adrian, Gough, Veronica, Bohning, Joel D., Liu, Siyan, Robinson-Hamm, Jacqueline N., Daniels, Heather, Majoros, William H., Devlin, Garth, Asokan, Aravind, Gersbach, Charles A.
Publicado 2021