title: Correction of Monogenic and Common Retinal Disorders with Gene Therapy.
abstract: 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.
authors.affiliation: Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, 10032 USA. js3899@cumc.columbia.edu.|Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, 10032 USA. js3899@cumc.columbia.edu.|State University of New York Downstate Medical Center, Brooklyn, NY, 11203 USA. js3899@cumc.columbia.edu.
authors.forename: Jesse D
authors.initials: JD
authors.lastname: Sengillo
authors.affiliation: Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, 10032 USA. sj2750@cumc.columbia.edu.|Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, 10032 USA. sj2750@cumc.columbia.edu.
authors.forename: Sally
authors.initials: S
authors.lastname: Justus
authors.affiliation: Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, 10032 USA. tc2803@cumc.columbia.edu.|Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, 10032 USA. tc2803@cumc.columbia.edu.|Department of Ophthalmology, Federal University of Espírito Santo, Vitória, 29075-910 Brazil. tc2803@cumc.columbia.edu.|Department of Ophthalmology, Federal University of São Paulo, São Paulo, 04021-001 Brazil. tc2803@cumc.columbia.edu.
authors.forename: Thiago
authors.initials: T
authors.lastname: Cabral
authors.affiliation: Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, 10032 USA. sht2@columbia.edu.|Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, 10032 USA. sht2@columbia.edu.|Department of Pathology & Cell Biology, Institute of Human Nutrition, College of Physicians and Surgeons, Columbia University, New York, NY, 10032 USA. sht2@columbia.edu.
authors.forename: Stephen H
authors.initials: SH
authors.lastname: Tsang
keywords: gene therapy, genome engineering, CRISPR, retinal degeneration, inherited retinal disease