Transplantation of human embryonic stem cells derived photoreceptors restores some visual function in Crx deficient mice

By D. Lamba, Jule Gust, T. Reh. Cell Stem Cell, 2009

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Summary Some of the most common causes of blindness involve the degeneration of photoreceptors in the neural retina; photoreceptor replacement therapy might restore some vision in these individuals. Embryonic stem (ES) cells could in principle provide a source of photoreceptors to repair the retina. We have previously shown that retinal progenitors can be efficiently derived from human ES cells. We now show that retinal cells derived from human ES cells will migrate into mouse retinas following intra-ocular injection, settle into the appropriate layers and express markers for differentiated cells, including both rod and cone photoreceptor cells. After transplantation of the cells into the subretinal space of adult Crx -/- mice (a model of Leber's Congenital Amaurosis), the hES cell derived retinal cells differentiate into functional photoreceptors and restore light responses to the animals. These results demonstrate that hES cells can, in principle, be used for photoreceptor replacement therapies.

Subfields: Cellular and Molecular Neuroscience, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/1c78d6b86bc2c723195de63a3f228bb7b25ca4e2

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