Retinitis Pigmentosa GTPase Regulator-related Retinopathy and Gene Therapy

Overview

Journal Saudi J Ophthalmol

Specialty Ophthalmology

Date 2023 Dec 29

PMID 38155670

Authors

Nida Wongchaisuwat

Alessia Amato

Andrew E Lamborn

Paul Yang

Lesley Everett

Mark E Pennesi

Affiliations

Soon will be listed here.

Abstract

Retinitis pigmentosa GTPase regulator -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 -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.

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