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Human Gene Therapy|June 17, 2015
Human α7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient MiceKristin N Heller, Chrystal L Montgomery, Kimberly M Shontz, et al.
Human Gene Therapy|December 22, 2020
Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular DystrophyDanielle A Griffin, Eric R Pozsgai, Kristin N Heller, et al.
Expert Opinion on Biological Therapy|February 8, 2020
Clinical development on the frontier: gene therapy for duchenne muscular dystrophyDamon R Asher, Khampaseuth Thapa, Sachi D Dharia, et al.
Human Gene Therapy|January 5, 2021
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular DystrophyRachael A Potter, Danielle A Griffin, Kristin N Heller, et al.
Molecular Therapy. Methods & Clinical Development|February 8, 2024
Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74Rachael A Potter, Ellyn L Peterson, Danielle Griffin, et al.
Journal of Market Access & Health Policy|June 1, 2023
Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United StatesAlexa C Klimchak, Lauren E Sedita, Louise R Rodino-Klapac, et al.
Journal of Translational Medicine|September 26, 2007
A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophyLouise R Rodino-Klapac, Paul M L Janssen, Chrystal L Montgomery, et al.
Molecular Therapy. Methods & Clinical Development|March 31, 2022
Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerationsJerry R Mendell, Anne M Connolly, Kelly J Lehman, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 29, 2013
AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathyZarife Sahenk, Gloria Galloway, Kelly Reed Clark, et al.
Human Gene Therapy|November 28, 2024
Long-Term Survival and Myocardial Function Following Systemic Delivery of Delandistrogene Moxeparvovec in DMDMDX RatsStephen Baine, Chris Wier, Luke Lemmerman, et al.
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