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FFB Written Articles » Macular Degeneration
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Researchers Target Innovative Stem Cell Treatment for AMD

An international research team has rescued vision in rats through transplantation of induced pluripotent stem cells or iPS — stem cells that are derived from mature cells or tissue that reside almost anywhere in the body. The team is working toward using this innovative approach to treat age-related macular degeneration (AMD), and potentially other retinal degenerative diseases, in humans. 

The research collaboration, which includes investigators from the University of California, Santa Barbara and University College London, used lung cells that were converted into stem cells by treating them with special proteins known as transcription factors. The stem cells were then manipulated forward to become retinal pigment epithelial (RPE) cells.

When the newly derived RPE cells were transplanted into rats with damaged RPE cells, the rats’ vision was retained. The rats that were not treated lost significant vision.

RPE cells play an essential supportive role in maintaining retinal health and vision, and are damaged in diseases such as age-related macular degeneration, Stargardt disease, and some forms of retinitis pigmentosa. In the study, the researchers verified that the transplanted RPE cells were in fact keeping photoreceptors, the cells responsible for vision, healthy and functioning.

The advantage of using induced pluripotent stem cells is that they have the properties of embryonic stem cells — they can be replicated and coaxed to become virtually any cell type in the body — but are not extracted from embryos. 

The research team will be evaluating induced pluripotent cells and other stem cell types in additional animal models of retinal degeneration with the goal of moving cell-based treatments into human trials.
 

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