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, Antoine Guyon, Joël Rousseau, Dominique L. Ouellet, Xavier Barbeau, Patrick Lagüe, 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.
Molecular Therapy · Aug 16, 2018Peer-reviewedMolecular Biology