Pluripotent stem cells (ES & iPS) for treatment of retinal dystrophies

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The impaired or complete loss of function of photoreceptor cells or supporting retinal pigmented epithelium (RPE) is the main cause of irreversible blindness in retinal diseases, such as inherited retinal degenerations and age-related macular degeneration (AMD). Rescuing the degenerated retina is a major challenge and cell replacement is one of the most promising approaches with the use of pluripotent stem cells. Human embryonic stem (ES) cells and induced pluripotent stem (iPS) cells that have the ability to be expanded indefinitely in culture whilst retaining their pluripotent status could be used as an unlimited source of retinal cells for tissue transplantation. The current challenge is actually to determine culture conditions for efficient and directed differentiation of human ES/ iPS cells into RPE or photoreceptors, so that large numbers of these cells might be transplanted at the optimal stage. We will present several approaches allowing the generation of RPE cells from human ES and iPS cells; and we will reported the ability of these stem cells to be committed into the neural retinal lineage, and further to be differentiated into photoreceptor cells following different protocols.

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