Title: | Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping |
Journal : | Molecular therapy. Methods & clinical development, 21 |
Authors: | Simmons TR ; Vetter TA ; Huang N ; Vulin-Chaffiol A ; Wein N ; Flanigan KM |
Material Type: | Article |
Publication Date: | 23/03/2021 |
Size: | p 325 |
Languages: | English |
Keywords: | Becker muscular dystrophy;Duchenne muscular dystrophy;U7snRNA;dystrophin;exon skipping;gene therapy; |
Pubmed / DOI : | Pubmed : 33898631 / DOI : 10.1016/j.omtm.2021.03.014 |
N° Profil MNM : | 2021051 |