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JCI Insight|May 5, 2017
MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophinKristin N Heller, Joshua T Mendell, Jerry R Mendell, et al.
Archives of Neurology|September 12, 2007
Gene therapy for duchenne muscular dystrophy: expectations and challengesLouise R Rodino-Klapac, Louis G Chicoine, Brian K Kaspar, et al.
Journal of Neuromuscular Diseases|November 19, 2016
Follistatin Gene Therapy Improves Ambulation in Becker Muscular DystrophySamiah A Al-Zaidy, Zarife Sahenk, Louise R Rodino-Klapac, et al.
Drug Metabolism and Disposition: the Biological Fate of Chemicals|November 8, 2024
Characterization of Nonclinical Drug Metabolism and Pharmacokinetic Properties of Phosphorodiamidate Morpholino Oligonucleotides, a Novel Drug Class for Duchenne Muscular DystrophyAndrew K L Goey, Marie Claire Mukashyaka, Yogesh Patel, et al.
Human Gene Therapy|March 28, 2017
Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by PrednisoneMegan L Cramer, Guohong Shao, Louise R Rodino-Klapac, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 13, 2017
Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E MiceEric R Pozsgai, Danielle A Griffin, Kristin N Heller, et al.
Biology Open|September 6, 2023
Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophyRachael A Potter, Danielle A Griffin, Kristin N Heller, et al.
Neurodegenerative Disease Management|September 2, 2021
Unmet needs and evolving treatment for limb girdle muscular dystrophiesEric Pozsgai, Danielle Griffin, Rachael Potter, et al.
Muscle & Nerve|February 12, 2009
Inhibition of myostatin with emphasis on follistatin as a therapy for muscle diseaseLouise R Rodino-Klapac, Amanda M Haidet, Janaiah Kota, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 16, 2013
AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic miceKristin N Heller, Chrystal L Montgomery, Paul Ml Janssen, et al.
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