Stem Cell Therapy for Inherited Retinal Disorders: Rebuilding the Retina.

Abstract

Inherited retinal disorders (IRDs) are a group of ophthalmic conditions that are characterized by progressive degeneration of photoreceptors and/or retinal pigment epithelium (RPE), leading to irreversible vision loss. IRDs are a promising target for stem cell therapies, which have largely aimed to restore vision by either replacement of degenerated photoreceptors or retinal pigment epithelial cells via transplantation of stem cells or preservation of cells through expression of neuroprotective factors. Several therapies have recently moved into early clinical trials, demonstrating that stem cell therapies can be safely administered through different routes of administration including intravitreal, sub-retinal, and suprachoroidal injection. However, further larger-scale studies are needed to demonstrate efficacy of these treatments for the prevention or reversal of vision loss from photoreceptor or RPE degeneration.