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Landrum Peay H, Fischer R, Tzeng JP, et al. Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy:…

Nelson CE, Wu Y, Gemberling MP, et al. Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy. Nature medicine…

Shimizu-Motohashi Y, Komaki H, Motohashi N, Takeda S, Yokota T, Aoki Y. Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current…

Nakamura A. Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy. Journal of personalized medicine 2019;9  …

Mingozzi F, High KA. Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood 2013;122:23-36   Abstract Gene…

Ertl HCJ, High KA. Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with…

Babacic H, Mehta A, Merkel O, Schoser B. CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: A systematic…

Amoasii L, Hildyard JCW, Li H, et al. Gene editing restores dystrophin expression in a canine model of Duchenne muscular…

Leborgne C, Latournerie V, Boutin S, et al. Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne…

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