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The New England Journal of Medicine|October 8, 2010
Dystrophin immunity in Duchenne's muscular dystrophyJerry R Mendell, Katherine Campbell, Louise Rodino-Klapac, et al.
Annals of Neurology|October 3, 2009
Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteinsJerry R Mendell, Louise R Rodino-Klapac, Xiomara Rosales-Quintero, 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.
The Journal of Clinical Investigation|March 24, 2007
Interplay of IKK/NF-kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophySwarnali Acharyya, S Armando Villalta, Nadine Bakkar, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 11, 2017
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional OutcomesJerry R Mendell, Zarife Sahenk, Samiah Al-Zaidy, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 23, 2013
Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogatesLouis G Chicoine, Louise R Rodino-Klapac, Guohong Shao, et al.
JAMA Neurology|June 16, 2020
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled TrialJerry R Mendell, Zarife Sahenk, Kelly Lehman, et al.
Medicine|July 3, 2019
Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophyLindsay N Alfano, Jay S Charleston, Anne M Connolly, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 18, 2014
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophyJerry R Mendell, Zarife Sahenk, Vinod Malik, et al.
Annals of Neurology|October 30, 2010
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2DJerry R Mendell, Louise R Rodino-Klapac, Xiomara Q Rosales, et al.
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