Jul 2026· American journal of ophthalmology-glaucoma· Vol 291, pp. 341-351· 0 citations· 60 references
Medicine
TL;DR
Inherited retinal disorders are a promising target for stem cell therapies, but 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.
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.
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Induced pluripotent stem cell (iPSC)-derived retinal organoids (ROs) have emerged as a platform for photoreceptor replacement therapy, supported by two complementary approaches: cone precursor cell transplantation and RO transplantation. Both have demonstrated proof of concept for functional synaptic integration with t...
Key translational and drug development challenges, including insufficient light sensitivity, interspecies differences, immune responses to viral vectors and microbial proteins, and the lack of standardized clinical endpoints for patients with profound visual impairment are discussed.
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