Questions Answered and Unanswered by the First CRISPR Editing Study in a Canine Model of Duchenne Muscular Dystrophy

• Published in: Human gene therapy Vol. 30; no. 5; p. 535
• Main Authors: Wasala, Nalinda B, Hakim, Chady H, Chen, Shi-Jie, Yang, N Nora, Duan, Dongsheng
• Format: Journal Article
• Language: English
• Published: United States 01.05.2019
• Subjects: Animals; CRISPR-Cas Systems; Dependovirus - genetics; Disease Models, Animal; Dogs; Gene Editing; Genetic Vectors - genetics; Humans; Muscular Dystrophy, Duchenne - genetics; Muscular Dystrophy, Duchenne - therapy; RNA, Guide, CRISPR-Cas Systems; CRISPR; DMD; dystrophin; dog; AAV; editing

Clustered regularly interspaced short palindromic repeats (CRISPR) editing is being considered as a potential gene repair therapy to treat Duchenne muscular dystrophy, a dystrophin-deficient lethal muscle disease affecting all muscles in the body. A recent preliminary study from the Olson laboratory...