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Stemming vision loss with stem cells
Valentina Marchetti, … , David F. Friedlander, Martin Friedlander
Valentina Marchetti, … , David F. Friedlander, Martin Friedlander
Published September 1, 2010
Citation Information: J Clin Invest. 2010;120(9):3012-3021. https://doi.org/10.1172/JCI42951.
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Review

Stemming vision loss with stem cells

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Abstract

Dramatic advances in the field of stem cell research have raised the possibility of using these cells to treat a variety of diseases. The eye is an excellent target organ for such cell-based therapeutics due to its ready accessibility, the prevalence of vasculo- and neurodegenerative diseases affecting vision, and the availability of animal models to demonstrate proof of concept. In fact, stem cell therapies have already been applied to the treatment of disease affecting the ocular surface, leading to preservation of vision. Diseases in the back of the eye, such as macular degeneration, diabetic retinopathy, and inherited retinal degenerations, present greater challenges, but rapidly emerging stem cell technologies hold the promise of autologous grafts to stabilize vision loss through cellular replacement or paracrine rescue effects.

Authors

Valentina Marchetti, Tim U. Krohne, David F. Friedlander, Martin Friedlander

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Figure 1

Schematic representation of the eye with images of diseases associated with different regions of the eye that may be amenable to treatment with stem cells.

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Schematic representation of the eye with images of diseases associated w...
As discussed in the text, the eye can be divided into anterior and posterior segments with different diseases characteristic of these segments. Diseases that would be potentially treated with stem cell–based therapies include corneal opacification (image reproduced with permission from the New England Journal of Medicine; ref. 9) and glaucoma (enlarged optic nerve head cup/disc ratio). Glaucoma is typically considered a disease of the anterior segment, since treatments have historically been directed at decreasing fluid production or increasing fluid outflow by targeting the ciliary body or TM, respectively. The vision-threatening pathology, however, is in the posterior segment principally affecting the ganglion cells and optic nerve. In the posterior segment, degenerative neuronal diseases could include those affecting photoreceptors such as retinitis pigmentosa and the photoreceptors and retinal pigmented epithelium such as atrophic macular degeneration. Retinal vascular diseases of the posterior segment would include DR and neovascular macular degeneration.

Copyright © 2025 American Society for Clinical Investigation
ISSN: 0021-9738 (print), 1558-8238 (online)

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