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December 8, 2025Molecular Medicine2 citationsOpen Access

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

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JMJessica Yuen Wuen MaMDMaciej DaniszewskiAPAlice Pébay

Key Points

  • Transplantation of retinal ganglion cells may improve vision loss caused by degeneration.
  • Key markers define retinal ganglion cells' identity and maturation stages during differentiation.
  • Analysis of human pluripotent stem cells shows their promise in drug screening for optic neuropathies.
  • These advancements highlight the potential of human-derived cells in regenerative medicine for vision restoration.

Abstract

Abstract 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.

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Cite This Study

Ma et al. (2025) studied this question.

synapsesocial.com/papers/69401f002d562116f28f9c68https://doi.org/10.1186/s10020-025-01405-0
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