1 Result(s)

Article

Transiently expressed CRISPR/Cas9 induces wild-type dystrophin in vitro in DMD patient myoblasts carrying duplications

Among the mutations arising in the DMD gene and causing Duchenne Muscular Dystrophy (DMD), 10–15% are multi-exon duplications. There are no current therapeutic approaches with the ability to excise large multi-ex...

Veronica Pini, Virginie Mariot, Julie Dumonceaux, John Counsell… in Scientific Reports (2022)