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Trends Mol Med. 2016 Feb;22(2):115-134. doi: 10.1016/j.molmed.2015.12.007. Epub 2016 Jan 11.

Cell-Based Therapy for Degenerative Retinal Disease.

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Rutgers New Jersey Medical School, Newark, NJ 07103, USA. Electronic address:


Stem cell-derived retinal pigment epithelium (RPE) and photoreceptors (PRs) have restored vision in preclinical models of human retinal degenerative disease. This review discusses characteristics of stem cell therapy in the eye and the challenges to clinical implementation that are being confronted today. Based on encouraging results from Phase I/II trials, the first Phase II clinical trials of stem cell-derived RPE transplantation are underway. PR transplant experiments have demonstrated restoration of visual function in preclinical models of retinitis pigmentosa and macular degeneration, but also indicate that no single approach is likely to succeed in overcoming PR loss in all cases. A greater understanding of the mechanisms controlling synapse formation as well as the immunoreactivity of transplanted retinal cells is urgently needed.

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