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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 3

Example of a potential treatment strategy for degenerative/dystrophic retinopathies using autologous iPSCs.

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Example of a potential treatment strategy for degenerative/dystrophic re...
Skin cells such as keratinocytes or fibroblasts are sampled from the patient by biopsy and expanded ex vivo. Reprogramming into iPSCs is achieved by conventional protocols using retroviral transfection or, preferentially, by alternative protocols that employ nonintegrating vectors or vector-free treatment with small molecules. Directed differentiation regimens are used to derive the required type of fully differentiated retinal cells, such as RPE or photoreceptors, from the iPSC cultures. Following further enrichment and expansion steps, retinal cells will be reimplanted directly into the subretinal space during intraocular surgery. By supplementing or replacing diseased cells, the transplanted cells may prevent further disease progression and visual decline. RPE cells generated from human 4-factor iPSCs have characteristic polygonal shapes, express ZO-1 junction proteins, and vectorially transport fluid, as evidenced by “dome” formation.

Copyright © 2023 American Society for Clinical Investigation
ISSN: 0021-9738 (print), 1558-8238 (online)

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