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Review
. 2018 Jan;13(1):89-96.
doi: 10.2217/rme-2017-0089. Epub 2018 Jan 23.

Autologous stem cell therapy for inherited and acquired retinal disease

Mary Ben L Apatoff  1   2 , Jesse D Sengillo  1   2   3 , Eugenia C White  2 , Mathieu F Bakhoum  2 , Alexander G Bassuk  4 , Vinit B Mahajan  5   6 , Stephen H Tsang  1   2   7   8
Affiliations
Review

Autologous stem cell therapy for inherited and acquired retinal disease

Mary Ben L Apatoff et al. Regen Med. 2018 Jan.

Abstract

The mammalian retina, derived from neural ectoderm, has little regenerative potential. For conditions where irreversible retinal pigment epithelium or photoreceptor cell loss occurs, advanced techniques are required to restore vision. Inherited retinal dystrophies and some acquired conditions, such as age-related macular degeneration, have a similar end result of photoreceptor cell death leading to debilitating vision loss. These diseases stand to benefit from future regenerative medicine as dietary recommendations and current pharmacologic therapy only seek to prevent further disease progression. Cell-based strategies, such as autologously derived induced pluripotent stem cells, have come a long way in overcoming previous technical and ethical concerns. Clinical trials for such techniques are already underway. These trials and the preceding preclinical studies will be discussed in the context of retinal disease.

Keywords: autologous; induced pluripotent stem cells; retinal degeneration; stem cell.

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Conflict of interest statement

Financial & competing interests disclosure

Jonas Children's Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory are supported by the National Institute of Health (5P30EY019007, R01EY018213, R01EY024698, R01EY026682 and R21AG050437), National Cancer Institute Core (5P30CA013696), the Research to Prevent Blindness (RPB) Physician-Scientist Award, unrestricted funds from RPB, New York, NY, USA. JD Sengillo is supported by the RPB Medical Student Eye Research Fellowship. SH Tsang is a member of the RD-CURE Consortium and is supported by the Tistou and Charlotte Kerstan Foundation, the Schneeweiss Stem Cell Fund, New York State (C029572), the Foundation Fighting Blindness New York Regional Research Center Grant (C-NY05-0705-0312), the Crowley Family Fund and the Gebroe Family Foundation. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

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