Gene and cell‐based therapies for inherited retinal disorders: An update
Year: 2016
Type: review
Abstract: 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.
DOI: 10.1002/ajmg.c.31534
Authors: Jesse D. Sengillo, Sally Justus, Yi‐Ting Tsai, Thiago Cabral, Stephen H. Tsang
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: green
Awards: Core Facilities for Vision Research, Evaluating GWAS AMD Candidate Loci by Gene Editing in Human iPS Cells, Mechanistic Studies on Regenerative Medicine Approaches to Childhood Blindness, Defining Barriers to Gene Therapy, Gene Editing and Silencing in Phototransduction, Cancer Center Support Grant
OpenAlex ID: W2556045776