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Genes 2017, 8(2), 53;

Correction of Monogenic and Common Retinal Disorders with Gene Therapy

Jonas Children’s Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, 10032 USA
Edward S. Harkness Eye Institute, New York‐Presbyterian Hospital, New York, NY, 10032 USA
State University of New York Downstate Medical Center, Brooklyn, NY, 11203 USA
Department of Ophthalmology, Federal University of Espírito Santo, Vitória, 29075‐910 Brazil
Department of Ophthalmology, Federal University of São Paulo, São Paulo, 04021‐001 Brazil
Department of Pathology & Cell Biology, Institute of Human Nutrition, College of Physicians and Surgeons, Columbia University, New York, NY, 10032 USA
Author to whom correspondence should be addressed.
Academic Editor: Selvarangan Ponnazhagan
Received: 1 November 2016 / Revised: 19 January 2017 / Accepted: 19 January 2017 / Published: 27 January 2017
(This article belongs to the Section Human Genomics and Genetic Diseases)
Full-Text   |   PDF [272 KB, uploaded 6 February 2017]


The past decade has seen major advances in gene‐based therapies, many of which show promise for translation to human disease. At the forefront of research in this field is ocular disease, as the eye lends itself to gene‐based interventions due to its accessibility, relatively immune‐privileged status, and ability to be non‐invasively monitored. A landmark study in 2001 demonstrating successful gene therapy in a large‐animal model for Leber congenital amaurosis set the stage for translation of these strategies from the bench to the bedside. Multiple clinical trials have since initiated for various retinal diseases, and further improvements in gene therapy techniques have engendered optimism for alleviating inherited blinding disorders. This article provides an overview of gene‐based strategies for retinal disease, current clinical trials that engage these strategies, and the latest techniques in genome engineering, which could serve as the next frontline of therapeutic interventions. View Full-Text
Keywords: gene therapy, genome engineering, CRISPR, retinal degeneration, inherited retinal disease gene therapy, genome engineering, CRISPR, retinal degeneration, inherited retinal disease
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).

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Sengillo, J.D.; Justus, S.; Cabral, T.; Tsang, S.H. Correction of Monogenic and Common Retinal Disorders with Gene Therapy. Genes 2017, 8, 53.

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