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

**Authors:** Jessica Yuen Wuen Ma, Maciej Daniszewski, Alice Pébay

PMC · DOI: 10.1186/s10020-025-01405-0 · Molecular Medicine · 2025-12-08

## TL;DR

This paper reviews how human stem cells can be used to create retinal ganglion cells for studying eye diseases and developing treatments.

## Contribution

The paper provides a comprehensive overview of recent advances in generating and using human retinal ganglion cells from stem cells.

## Key findings

- Human pluripotent stem cells can be differentiated into retinal ganglion cells for disease modeling.
- Recent methods improve the functional characterization of these cells for translational applications.
- These cells offer potential for drug screening and cell replacement therapies in optic neuropathies.

## 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.

## Linked entities

- **Diseases:** glaucoma (MONDO:0005041)

## Full-text entities

- **Diseases:** Retinal (MESH:D012173), vision loss (MESH:D014786), glaucoma (MESH:D005901), optic neuropathies (MESH:D009901)
- **Species:** Homo sapiens (human, species) [taxon 9606]

## Full text

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## Figures

3 figures with captions in the complete paper: https://tomesphere.com/paper/PMC12911366/full.md

## References

3 references — full list in the complete paper: https://tomesphere.com/paper/PMC12911366/full.md

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Source: https://tomesphere.com/paper/PMC12911366