Induced pluripotent stem cell therapies for retinal disease

Abstract

This review will discuss how recent advances with induced pluripotent stem (iPS) cells have brought the science of stem cell biology much closer to clinical application for patients with retinal degeneration. The ability to generate embryonic stem cells by reprogramming DNA taken from adult cells was demonstrated by the cloning of Dolly, the sheep, by somatic cell nuclear transfer, over 10 years ago. Recently, it has been shown that adult cells can be reprogrammed directly, without the need for a surrogate oocyte, through the generation of iPS cells. The method of reprogramming has since been optimized to avoid the use of retroviruses, making the process considerably safer. Last year, human iPS cells were isolated from an 80-year-old patient with neurodegenerative disease and differentiated into neurons in vitro. For stem cell therapies, the retina has the optimal combination of ease of surgical access, combined with an ability to observe transplanted cells directly through the clear ocular media. The question now is which retinal diseases are most appropriate targets for clinical trials using iPS cell approaches.