Retinal ganglion cells (RGCs) are highly specialized neuronal cells located in the innermost layer of the retina and serve to relay visual information to the brain, with their axons collectively forming the optic nerve. Loss or damage to the RGCs results in visual impairment and ultimately blindness. Several diseases affect the RGCs exclusively, the most common being glaucoma. Even though the mechanisms of glaucoma are not fully understood, some common treatments can delay cell death. Pharmacological intervention or laser therapy is thought to reduce the intraocular pressure and therefore reduce cell damage, but ultimately these therapies are often transient and stop working. Additional strategies to rescue RGCs and prevent their loss are being explored. Human pluripotent stem cells (hPSCs), including both embryonic and induced pluripotent stem cells, serve as an effective in vitro model of retinal development and repair. In addition, RGCs differentiated from hPSCs can also provide an unlimited source of transplantable cells for use in cell replacement strategies. This review addresses some of the technical and clinical issues and concerns with generating bone fide RGCs in vitro. We also outline the potential applications of hPSC-derived RGCs as a useful tool in disease modeling and drug screening in order to advance knowledge of optic neuropathies.
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