Gene and cell‐based therapies for inherited retinal disorders: An update

JD Sengillo, S Justus, YT Tsai… - American Journal of …, 2016 - Wiley Online Library
JD Sengillo, S Justus, YT Tsai, T Cabral, SH Tsang
American Journal of Medical Genetics Part C: Seminars in Medical …, 2016Wiley Online Library
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 …
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. © 2016 Wiley Periodicals, Inc.
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