Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated <i>DMD</i> exon 2 skipping.

Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping.

Simmons, Tabatha R; Vetter, Tatyana A; Huang, Nianyuan; Vulin-Chaffiol, Adeline; Wein, Nicolas; Flanigan, Kevin M.

Simmons TR; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Vetter TA; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Huang N; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Vulin-Chaffiol A; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Wein N; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Flanigan KM; Department of Pediatrics, Ohio State University, Columbus, OH, USA.

Mol Ther Methods Clin Dev ; 21: 325-340, 2021 Jun 11.

Article en En | MEDLINE | ID: mdl-33898631