Maximizing functional photoreceptor differentiation from adult human retinal stem cells.

Maximizing functional photoreceptor differentiation from adult human retinal stem cells.
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DOI:
10.1002/stem.279
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发表时间:
2010-03-31
期刊:
影响因子:
5.2
通讯作者:
van der Kooy, Derek
van der Kooy, Derek
中科院分区:
医学2区
文献类型:
--
作者:
Inoue, Tomoyuki;Coles, Brenda L. K.;Dorval, Kim;Bremner, Rod;Bessho, Yasumasa;Kageyama, Ryoichiro;Hino, Shinjiro;Matsuoka, Masao;Craft, Cheryl M.;McInnes, Roderick R.;Tremblay, Francois;Prusky, Glen T.;van der Kooy, Derek

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Retinal stem cells (RSCs) are present in the ciliary margin of the adult human eye and can give rise to all retinal cell types. Here we show that modulation of retinal transcription factor gene expression in human RSCs greatly enriches photoreceptor progeny, and that strong enrichment was obtained with the combined transduction of OTX2 and CRX together with the modulation of CHX10. When these genetically modified human RSC progeny are transplanted into mouse eyes, their retinal integration and differentiation is superior to unmodified RSC progeny. Moreover, electrophysiologic and behavioral tests show that these transplanted cells promote functional recovery in transducin mutant mice. This study suggests that gene modulation in human RSCs may provide a source of photoreceptor cells for the treatment of photoreceptor disease.
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