Abstract
Patient-derived human-induced pluripotent stem cells (iPSCs) have been critical in advancing our understanding of the underlying mechanisms of numerous retinal disorders. Many of these retinal disorders have no effective treatment and result in severe visual impairment and even blindness. Among the retinal degenerative diseases modeled by iPSCs are age-related macular degeneration (AMD), glaucoma, Leber congenital amaurosis (LCA), retinitis pigmentosa (RP), and autosomal dominant retinitis pigmentosa (adRP). In addition to studying retinal disease ontogenesis and pathology, hiPSCs have clinical and pharmacological applications, such as developing drug screening and gene therapy approaches and new cell-based clinical treatments. Recent studies have primarily focused on three retinal cell fates- retinal pigmented epithelium cells (RPE), retinal ganglion cells (RGCs), and photoreceptor cells- and have demonstrated that hiPSCs have great potential for increasing our knowledge of and developing treatments for retinal degenerative disorders.
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