Single Center Experience with Voretigene Neparvovec Gene Augmentation Therapy in RPE65 Mutation–Associated Inherited Retinal Degeneration in a Clinical Setting-Reference-Cited by-同舟云学术

Single Center Experience with Voretigene Neparvovec Gene Augmentation Therapy in RPE65 Mutation–Associated Inherited Retinal Degeneration in a Clinical Setting

Published:2024-02 Issue:2 Volume:131 Page:161-178
ISSN:0161-6420
Container-title:Ophthalmology
language:en
Short-container-title:Ophthalmology

Author:

Lorenz Birgit^ORCID,Künzel Sandrine H.,Preising Markus N.,Scholz Johanna P.,Chang Petrus,Holz Frank G.,Herrmann Philipp

Funder

Novartis International AG

Novartis Pharma GmbH

Deutsche Ophthalmologische Gesellschaft

University of Bonn

Publisher

Elsevier BV

Subject

Ophthalmology

Reference47 articles.

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3. Current management of patients with RPE65 mutation-associated inherited retinal degenerations in Europe: results of a multinational survey by the European Vision Institute Clinical Research Network;Lorenz;Ophthalmic Res,2021

4. Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle;Redmond;Proc Natl Acad Sci U S A,2005

5. A comprehensive clinical and biochemical functional study of a novel RPE65 hypomorphic mutation;Lorenz;Invest Ophthalmol Vis Sci,2008

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1. Frequency and Genetic Spectrum of Inherited Retinal Dystrophies in a Large Dutch Pediatric Cohort: The RD5000 Consortium;Investigative Ophthalmology & Visual Science;2024-08-29

2. Objective Outcomes to Evaluate Voretigene Neparvovec Treatment Effects in Clinical Practice;Ophthalmology Retina;2024-07

3. Enhancing pediatric access to cell and gene therapies;Nature Medicine;2024-06-17

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