Abstract

Stem cells are a promising source of cellular material for treating a variety of retinal diseases. Already several clinical trials have been approved, and more are being developed. The stem cell types most frequently employed, and that will be most extensively explored in this chapter, are human embryonic stem cells (hESCs) and induced pluripotent stem cells (hiPSCs). In particular, retinal pigment epithelium (RPE) and photoreceptors derived from hESCs and hiPSCs have been investigated as a treatment for retinal diseases associated with the degeneration of these cell types, such as age-related macular degeneration (AMD), Stargardt disease, and retinitis pigmentosa. Additionally, hiPSC-derived vascular progenitor (VP) cells or endothelial progenitor cells are gaining more attention for the treatment of dry AMD and diabetic and ischemic retinopathy. While significant progress has been made using these cells in translating basic scientific investigations to clinical trials in patients, major regulatory, safety, and technical challenges remain to be overcome before such stem cell–based retinal therapies become standard.

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