Journal
AMERICAN JOURNAL OF MEDICAL GENETICS PART C-SEMINARS IN MEDICAL GENETICS
Volume 172, Issue 4, Pages 349-366Publisher
WILEY
DOI: 10.1002/ajmg.c.31534
Keywords
retinal degeneration; gene therapy; stem cells; iPSCs; CRISPR; leber congenital amaurosis; choroideremia; retinitis pigmentosa; Usher syndrome; retinoschisis; leber hereditary optic neuropathy; achromatopsia
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Funding
- National Institute of Health [5P30EY019007, R01EY018213, R01EY024698, R01EY026682, R21AG050437]
- National Cancer Institute Core [5P30CA013696]
- Research to Prevent Blindness (RPB) Physician-Scientist Award
- RPB, New York, NY, USA
- RPB medical student fellowship
- International Council of Ophthalmology-Retina Research Foundation Helmerich Fellowship
- Tistou and Charlotte Kerstan Foundation
- Schneeweiss Stem Cell Fund, New York State [C029572]
- Foundation Fighting Blindness New York Regional Research Center Grant [C-NY05-0705-0312]
- Joel Hoffman Fund
- Professor Gertrude Rothschild Stem Cell Foundation
- Gebroe Family Foundation
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Retinal degenerations present a unique challenge as disease progression is irreversible and the retina has little regenerative potential. No current treatments for inherited retinal disease have the ability to reverse blindness, and current dietary supplement recommendations only delay disease progression with varied results. However, the retina is anatomically accessible and capable of being monitored at high resolution in vivo. This, in addition to the immune-privileged status of the eye, has put ocular disease at the forefront of advances in gene- and cell-based therapies. This review provides an update on gene therapies and randomized control trials for inherited retinal disease, including Leber congenital amaurosis, choroideremia, retinitis pigmentosa, Usher syndrome, X-linked retinoschisis, Leber hereditary optic neuropathy, and achromatopsia. New gene-modifying and cell-based strategies are also discussed. (c) 2016 Wiley Periodicals, Inc.
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