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Human Molecular Genetics|February 26, 2016
Defective membrane fusion and repair in Anoctamin5-deficient muscular dystrophyDanielle A Griffin, Ryan W Johnson, Jarred M Whitlock, et al.
Muscle & Nerve|June 4, 2026
Five-Year Outcomes With Delandistrogene Moxeparvovec in Patients With Duchenne Muscular Dystrophy: A Phase 1/2a StudyJerry R Mendell, Zarife Sahenk, Linda P Lowes, et al.
The American Journal of Pathology|September 7, 2011
mdx(⁵cv) mice manifest more severe muscle dysfunction and diaphragm force deficits than do mdx MiceNicholas Beastrom, Haiyan Lu, Allison Macke, et al.
Plos One|June 22, 2012
Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transferWilliam E Grose, K Reed Clark, Danielle Griffin, et al.
Molecular Therapy. Methods & Clinical Development|February 2, 2018
Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHDLindsay M Wallace, Nizar Y Saad, Nettie K Pyne, et al.
American Journal of Physiology. Cell Physiology|December 26, 2008
Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type micePaul T Martin, Rui Xu, Louise R Rodino-Klapac, et al.
Molecular Therapy. Methods & Clinical Development|February 23, 2023
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse modelYoung-Eun Seo, Stephen H Baine, Amber N Kempton, et al.
Muscle & Nerve|April 5, 2013
Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulationXiomara Q Rosales, Vinod Malik, Amita Sneh, et al.
Human Molecular Genetics|July 19, 2013
Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD modelLouise R Rodino-Klapac, Paul M L Janssen, Kimberly M Shontz, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 12, 2009
Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular deliveryLouise R Rodino-Klapac, Chrystal L Montgomery, William G Bremer, et al.
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