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Mendell JR, Sahenk Z, Lehman K, et al. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy:…

Wasala NB, Hakim CH, Chen SJ, Yang NN, Duan D. Questions Answered and Unanswered by the First CRISPR Editing Study…

Asher DR, Thapa K, Dharia SD, et al. Clinical development on the frontier: gene therapy for duchenne muscular dystrophy. Expert…

Jones D. Duchenne muscular dystrophy awaits gene therapy. Nature biotechnology 2019;37:335-337

Song Y, Morales L, Malik AS, et al. Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models.…

Duan D. Micro-utrophin Therapy for Duchenne Muscular Dystrophy. Molecular therapy : the journal of the American Society of Gene Therapy…

Crudele JM, Chamberlain JS. AAV-based gene therapies for the muscular dystrophies. Human molecular genetics 2019;28:R102-R107   Abstract Muscular dystrophy is…

Xu L, Lau YS, Gao Y, Li H, Han R. Life-Long AAV-Mediated CRISPR Genome Editing in Dystrophic Heart Improves Cardiomyopathy…

Cai A, Kong X. Development of CRISPR-Mediated Systems in the Study of Duchenne Muscular Dystrophy. Human gene therapy methods 2019;30:71-80…

Paquin RS, Fischer R, Mansfield C, et al. Priorities when deciding on participation in early-phase gene therapy trials for Duchenne…