Abstract

Loss of the light-sensing photoreceptors results in vision impairment and blindness, representing one of the main causes for disability in industrialized societies. Although the pathophysiology of diseases such as age-related macular degeneration, retinitis pigmentosa, or cone-rod dystrophies is highly heterogeneous, the final endpoint of such conditions is the degeneration of photoreceptors with no established therapies currently available for their replacement. However, recent preclinical studies demonstrated the feasibility of cell transplantation into the adult mammalian retina. Indeed, young post-mitotic photoreceptors represent a suitable cell type for such replacement approach as they showed proper integration and maturation following grafting. Importantly, donor photoreceptors connected to endogenous neurons allowing some functional rescue in mouse models of night blindness. Furthermore, methods for the generation of transplantable photoreceptors from pluripotent stem cells in vitro have been established. Here, we will review recent developments in the field of photoreceptor transplantation and will discuss still existing roadblocks toward clinical application.

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