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Willcocks RJ, Forbes SC, Walter GA, et al. Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With…

Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, et al. Current Clinical Applications of in vivo Gene Therapy with AAVs. Molecular therapy…

Chiu W, Hsun YH, Chang KJ, et al. Current Genetic Survey and Potential Gene-Targeting Therapeutics for Neuromuscular Diseases. International journal…

Brenner D, Ludolph AC, Weishaupt JH. Gene specific therapies - the next therapeutic milestone in neurology. Neurol Res Pract 2020;2:25…

Buscara L, Gross DA, Daniele N. Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to…

Sharma G, Sharma AR, Bhattacharya M, Lee SS, Chakraborty C. CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of…

Heslop E, Turner C, Irvin A, Muntoni F, Straub V, Guglieri M. Gene therapy in Duchenne muscular dystrophy: Identifying and…

McKim DA, Cripe TP, Cripe LH. The effect of emerging molecular and genetic therapies on cardiopulmonary disease in Duchenne muscular…

Schneider AE, Aartsma-Rus A. Developments in reading frame restoring therapy approaches for Duchenne muscular dystrophy. Expert Opin Biol Ther 2020:1-17…

Abreu NJ, Waldrop MA. Overview of Gene Therapy in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy. Pediatr Pulm. 2020  …