Rachael A. Potter

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman et al. · JAMA Neurology · 2020 · 392 citations · Full text

Pharmacy, Pediatrics, Pharmacotherapy +17

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald et al. · Nature Medicine · 2024 · 157 citations · Full text

Medicine, Molecular Genetics, Molecular Medicine +15

Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to &lt;8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study <scp>SRP</scp>‐9001‐103 (<scp>ENDEAVOR</scp>)

Craig M. Zaidman, Crystal M. Proud, Craig M. McDonald et al. · Annals of Neurology · 2023 · 91 citations · Full text

Medicine, Molecular Medicine, Translational Sciences +10

Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy

Rachael A. Potter, Danielle A. Griffin, Patricia Sondergaard et al. · Human Gene Therapy · 2017 · 86 citations · Full text

Medicine, Molecular Biology, Molecular Medicine +16

Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the <i>mdx</i> Mouse Model of Duchenne Muscular Dystrophy

Rachael A. Potter, Danielle A. Griffin, Kristin N. Heller et al. · Human Gene Therapy · 2021 · 85 citations · Full text

Medicine, Molecular Biology, Molecular Genetics +17