Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
2017 · New England Journal of Medicine · 2,229 citations
https://doi.org/10.1056/nejmoa1706198
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Jerry R. Mendell
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Jerry R. Mendell is an academic researcher from Nationwide Children's Hospital. The author has contributed to research in topics: Neurogenetic and Muscular Disorders Research & RNA modifications and cancer & Peripheral Neuropathies and Disorders. The author has an h-index of 66, co-authored 247 publications.
ORCID: 0000-0002-2691-0200Papers by this author
worksCurrent Clinical Applications of In Vivo Gene Therapy with AAVs
2020 · Molecular Therapy · 703 citations
https://doi.org/10.1016/j.ymthe.2020.12.007
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
2021 · The Lancet Neurology · 449 citations
https://doi.org/10.1016/s1474-4422(21)00001-6
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial
2022 · Nature Medicine · 273 citations
https://doi.org/10.1038/s41591-022-01866-4
Painful Sensory Neuropathy
2003 · New England Journal of Medicine · 266 citations
https://doi.org/10.1056/nejmcp022282
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
2022 · Nature Medicine · 255 citations
https://doi.org/10.1038/s41591-022-01867-3
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