Human pluripotent stem cell-derived retinal ganglion cells: advances in differentiation and translational applications

Retinal ganglion cells (RGCs) are neurons that transmit visual information from the retina to the brain. Their degeneration, as seen in glaucoma and other optic neuropathies, leads to irreversible vision loss. As mature human RGCs are difficult to access, most of their studies rely on rodent models, which do not fully recapitulate human retinal biology. Human pluripotent stem cells (hPSCs) provide a promising source for generating RGCs in vitro, supporting disease modelling, drug screening, and future cell replacement therapies. This review outlines key markers that define RGC identity, maturation stages, and subtype diversity. We summarise recent advances in the differentiation of hPSCs towards RGCs, their functional characterisation, and their applications in disease modelling, drug screening, and transplantation.