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Molecular Therapy : the Journal of the American Society of Gene Therapy|December 14, 2020
Current Clinical Applications of In Vivo Gene Therapy with AAVsJerry R Mendell, Samiah A Al-Zaidy, Louise R Rodino-Klapac, et al.Neurology|May 13, 2018
Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin productionJay S Charleston, Frederick J Schnell, Johannes Dworzak, et al.Annals of Clinical and Translational Neurology|March 28, 2015
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal ModelsPatricia C Sondergaard, Danielle A Griffin, Eric R Pozsgai, et al.Scientific Reports|January 3, 2025
Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapyRachael A Potter, Ida H Moeller, Sohrab Khan, et al.Human Gene Therapy|July 15, 2017
Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for DysferlinopathyRachael A Potter, Danielle A Griffin, Patricia C Sondergaard, et al.JAMA Neurology|May 12, 2025
Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy: An Exploratory Analysis From the EMBARK Randomized Clinical TrialKrista Vandenborne, Glenn A Walter, Volker Straub, et al.Nature Medicine|January 4, 2024
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial resultsJerry R Mendell, Eric R Pozsgai, Sarah Lewis, et al.Muscle & Nerve|August 14, 2023
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trialJerry R Mendell, Zarife Sahenk, Kelly J Lehman, et al.Human Gene Therapy|March 7, 2019
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb InfusionJerry R Mendell, Louis G Chicoine, Samiah A Al-Zaidy, et al.Science Translational Medicine|April 7, 2010
Follistatin gene delivery enhances muscle growth and strength in nonhuman primatesJanaiah Kota, Chalonda R Handy, Amanda M Haidet, et al.Pageof 7