Retinitis pigmentosa GTPase regulator‑related retinopathy and gene therapy
2
Issued Date
2023-10-01
Resource Type
ISSN
13194534
eISSN
25426680
Scopus ID
2-s2.0-85182577233
Journal Title
Saudi Journal of Ophthalmology
Volume
37
Issue
4
Start Page
276
End Page
286
Rights Holder(s)
SCOPUS
Bibliographic Citation
Saudi Journal of Ophthalmology Vol.37 No.4 (2023) , 276-286
Suggested Citation
Wongchaisuwat N., Amato A., Lamborn A.E., Yang P., Everett L., Pennesi M.E. Retinitis pigmentosa GTPase regulator‑related retinopathy and gene therapy. Saudi Journal of Ophthalmology Vol.37 No.4 (2023) , 276-286. 286. doi:10.4103/sjopt.sjopt_168_23 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/95802
Title
Retinitis pigmentosa GTPase regulator‑related retinopathy and gene therapy
Author's Affiliation
Corresponding Author(s)
Other Contributor(s)
Abstract
Retinitis pigmentosa GTPase regulator (RPGR)‑related retinopathy is a retinal dystrophy inherited in a X‑linked recessive manner that typically causes progressive visual loss starting in childhood with severe visual impairment by the fourth decade of life. It manifests as an early onset and severe form of retinitis pigmentosa. There are currently no effective treatments for RPGR‑related retinopathy; however, there are multiple clinical trials in progress exploring gene augmentation therapy aimed at slowing down or halting the progression of disease and possibly restoring visual function. This review focuses on the molecular biology, clinical manifestations, and the recent progress of gene therapy clinical trials.