Molecular BiologyMolecular TherapyPeer-reviewed
CRISPR-induced deletion with SaCas9 restores dystrophin expression in dystrophic models in vitro and in vivo.
Benjamin L. Duchêne, Khadija Cherif, Jean-Paul Iyombe-Engembe…·Jacques P. Tremblay
This study demonstrates that SaCas9-mediated deletion restores dystrophin expression in DMD patient-derived myoblasts and in vivo in a mouse model.