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The Promise of Pluripotent Stem Cells for Age Related Macular Degeneration

Jun 30, 2014 1:00:47 PM / by Shweta

Pluripotent stem cells offer a new prototype for Age Related Macular Degeneration modeling and therapies.

Age-related macular Degeneration (AMD) is a eye disease characterized by degeneration of the photoreceptors in the macula, the central part of the eye. It is a one of the leading cause of the blindness in people over age 55 in the U.S. and the developed world. This condition causes impairment of central visual acuity. In normal conditions, photoreceptor cells of retina are supported metabolically and structurally by a layer of cells called Retinal Pigment Epithelial (RPE) Cells. In absence of RPE cells, photoreceptor cells degenerate and die, which is an early and critical sign of AMD. Thus, replacing dead or dying RPE cells in AMD could be a way to slow the disease process and even improve vision[1].

Pluripotent stem cells (PSCs), by definition, can give rise to any type of cell in the body. In one of our previous blogs we have described that patient-specific pluripotent stem cells can provide better therapeutic options for patients suffering with diabetes mellitus. Recently, it has been documented that human embryonic stem cells (HESCs) and human induced pluripotent stem cells (iPSCs) can differentiate into retinal progenitor and RPE cells. These differentiated cells mimic to human native RPE cells in terms of their ability to differentiate into functional RPE cells. Creation of induced pluripotent stem cells derived from AMD patients provide unique advantages to understand the mechanism that initiate and drive the early events of disease. This approach will help in understanding the role of specific genes in AMD pathogenesis.

Treating macular degeneration with a patient’s own induced pluripotent stem cells Treating macular degeneration with a patient’s own induced pluripotent stem cells. Image

HESC-derived RPE cells have been shown to safely integrate in the human eye and are currently in a phase I/IIa clinical trial in humans for AMD. Four months post-transplantation, hESC-derived RPE cells had survived and were not rejected. They integrated well and did not give any adverse effects (such as tumor formation, hyperproliferation, or ectopic growth). In some cases visual improvement was also noted. Recently, the clinical trial for induced pluripotent stem cells -RPE cells were approved for AMD patients  in Japan (RIKEN). Identification of the disease related markers and therapeutic response of an in vitro disease model of AMD will further improve the therapeutic efficacy to treat AMD.

It has been established now that pluripotent stem cells have tremendous therapeutic potential for several diseases. If you need customized stem cells with established protocols for your research, or for cell therapy visit HemaCare, a leading provider of stem cells.


1. Falkner-Radler CI, Krebs I, Glittenberg C, Povazay B, Drexler W, Graf A, Binder S. Human retinal pigment epithelium (RPE) transplantation: outcome after autologous RPE-choroid sheet and RPE cell-suspension in a randomised clinical study. Br J Ophthalmol 2011;95:370-5.

2. Davidson KC, Guymer RH, Pera MF, Pébay A. Human Pluripotent Stem Cell Strategies for Age-Related Macular Degeneration. Optom Vis Sci. 2014 May 22.






Topics: aging, cellular reprogramming, iPSCs, Stem Cells, tissue engineering, Basic Research


Written by Shweta

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