Publications by authors named "Sarah McCague"

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Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.
Kiran Musunuru, Sarah A Grandinette, Xiao Wang, Taylor R Hudson, Kevin Briseno, Anne Marie Berry, Julia L Hacker, Alvin Hsu, Rachel A Silverstein, Logan T Hille

June 2025

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Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns.
Dennis J Keselman, Juliana C Small, Tiffany Seneviratne, Sarah McCague, Taylor L Kaschak

Mol Ther· October 2025

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Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial.
Tomas S Aleman, Rachel M Huckfeldt, Leona W Serrano, Denise J Pearson, Grace K Vergilio, Sarah McCague

Ophthalmology· October 2022

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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.
Albert M Maguire, Stephen Russell, Jennifer A Wellman, Daniel C Chung, Zi-Fan Yu, Sarah McCague

Ophthalmology· September 2019

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The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene.
Daniel C Chung, Mette Bertelsen, Birgit Lorenz, Mark E Pennesi, Bart P Leroy, Sarah McCague

Am J Ophthalmol· March 2019

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Novel mobility test to assess functional vision in patients with inherited retinal dystrophies.
Daniel C Chung, Sarah McCague, Zi-Fan Yu, Satha Thill, Julie DiStefano-Pappas

Clin Exp Ophthalmol· April 2018

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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Stephen Russell, Jean Bennett, Jennifer A Wellman, Daniel C Chung, Zi-Fan Yu, Sarah McCague

Lancet· August 2017

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Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.
Jean Bennett, Jennifer Wellman, Kathleen A Marshall, Sarah McCague, Manzar Ashtari

Lancet· August 2016

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AAV2 gene therapy readministration in three adults with congenital blindness.
Jean Bennett, Manzar Ashtari, Jennifer Wellman, Kathleen A Marshall, Laura L Cyckowski, Sarah McCague

Sci Transl Med· February 2012

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