Comparison of dystrophin expression following gene editing and gene replacement in an aged preclinical DMD animal model

• Published in: Molecular therapy Vol. 30; no. 6; pp. 2176 - 2185
• Main Authors: Bengtsson, Niclas E., Crudele, Julie M., Klaiman, Jordan M., Halbert, Christine L., Hauschka, Stephen D., Chamberlain, Jeffrey S.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.06.2022; American Society of Gene & Cell Therapy
• Subjects: AAV; Aging; Animals; Cas9; CRISPR; CRISPR-Cas Systems; CXMD; Disease Models, Animal; Disease Progression; DMD; Dogs; Duchenne; Dystrophin - genetics; gene editing; Gene Editing - methods; micro-dystrophin; muscle; Muscle, Skeletal - metabolism; Muscular Dystrophy, Duchenne - genetics; Muscular Dystrophy, Duchenne - therapy; Original; CRISPR; CXMD; micro-dystrophin; AAV; Duchenne; muscle; dogs; DMD; Cas9; gene editing

Gene editing has shown promise for correcting or bypassing dystrophin mutations in Duchenne muscular dystrophy (DMD). However, preclinical studies have focused on young animals with limited muscle fibrosis and wasting, thereby favoring muscle transduction, myonuclear editing, and prevention of disea...