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J. Clin. Med. 2015, 4(4), 567-578;

Patient-Specific iPSC-Derived RPE for Modeling of Retinal Diseases

College of Physicians and Surgeons, Columbia University, 100 Haven Ave, Apt 14B, New York, NY 10032, USA
Department of Ophthalmology, Columbia University, 635 W 165th St, New York, NY 10032, USA
Author to whom correspondence should be addressed.
Academic Editor: Michael J. Edel
Received: 17 September 2014 / Revised: 26 February 2015 / Accepted: 3 March 2015 / Published: 31 March 2015
(This article belongs to the Special Issue iPS Cells for Modelling and Treatment of Human Diseases)
Full-Text   |   PDF [61 KB, uploaded 31 March 2015]


Inherited retinal diseases, such as age-related macular degeneration and retinitis pigmentosa, are the leading cause of blindness in the developed world. Currently, treatments for these conditions are limited. Recently, considerable attention has been given to the possibility of using patient-specific induced pluripotent stem cells (iPSCs) as a treatment for these conditions. iPSCs reprogrammed from adult somatic cells offer the possibility of generating patient-specific cell lines in vitro. In this review, we will discuss the current literature pertaining to iPSC modeling of retinal disease, gene therapy of iPSC-derived retinal pigmented epithelium (RPE) cells, and retinal transplantation. We will focus on the use of iPSCs created from patients with inherited eye diseases for testing the efficacy of gene or drug-based therapies, elucidating previously unknown mechanisms and pathways of disease, and as a source of autologous cells for cell replacement. View Full-Text
Keywords: retinal disease; induced pluripotent stem cells; gene therapy retinal disease; induced pluripotent stem cells; gene therapy
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).

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Nguyen, H.V.; Li, Y.; Tsang, S.H. Patient-Specific iPSC-Derived RPE for Modeling of Retinal Diseases. J. Clin. Med. 2015, 4, 567-578.

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