Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
Titel:
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
Auteur:
Wein, Nicolas Vetter, Tatyana A. Vulin, Adeline Simmons, Tabatha R. Frair, Emma C. Bradley, Adrienne J. Gushchina, Liubov V. Almeida, Camila F. Huang, Nianyuan Lesman, Daniel Rajakumar, Dhanarajan Weiss, Robert B. Flanigan, Kevin M.