Fellowship · Columbus, OH
Sponsored by Nationwide Children's Hospital
Connecting copper and cancer: from transition metal signalling to metalloplasia.
Nature reviews. Cancer · 2022 · 1,024 citations · Stephen Kaler
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.
Nature medicine · 2025 · 143 citations · Jerry R. Mendell
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Molecular therapy : the journal of the American Society of Gene Therapy · 2021 · 578 citations · Jerry R. Mendell
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.
The Lancet. Neurology · 2021 · 399 citations · Anne Connolly, Jerry R. Mendell
Current clinical applications of AAV-mediated gene therapy.
Molecular therapy : the journal of the American Society of Gene Therapy · 2025 · 87 citations · Kevin Flanigan, Megan Waldrop
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.
Nature medicine · 2022 · 263 citations · Jerry R. Mendell
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.
Nature medicine · 2022 · 236 citations · Jerry R. Mendell
Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study).
Annals of neurology · 2021 · 248 citations · Chad Hoyle