用于视网膜疾病的干细胞疗法：重现发育过程以替代退化细胞。
Stem cell therapies for retinal diseases: recapitulating development to replace degenerated cells.
作者信息
Zhao Cuiping, Wang Qingjie, Temple Sally
机构信息
Neural Stem Cell Institute, 1 Discovery Drive, Rensselaer, NY 12144, USA.
Neural Stem Cell Institute, 1 Discovery Drive, Rensselaer, NY 12144, USA
出版信息
Development. 2017 Apr 15;144(8):1368-1381. doi: 10.1242/dev.133108.
Abstract
Retinal degenerative diseases are the leading causes of blindness worldwide. Replacing lost retinal cells via stem cell-based therapies is an exciting, rapidly advancing area of translational research that has already entered the clinic. Here, we review the status of these clinical efforts for several significant retinal diseases, describe the challenges involved and discuss how basic developmental studies have contributed to and are needed to advance clinical goals.
摘要
视网膜退行性疾病是全球失明的主要原因。通过基于干细胞的疗法替代丢失的视网膜细胞是一个令人兴奋且发展迅速的转化研究领域，该领域已进入临床阶段。在此，我们综述了针对几种重要视网膜疾病的这些临床研究进展，描述了其中涉及的挑战，并讨论了基础发育研究如何推动了临床目标以及为实现临床目标还需要哪些基础发育研究。
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本文引用的文献
1
Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration.
N Engl J Med. 2017 Mar 16;376(11):1038-1046. doi: 10.1056/NEJMoa1608368.
2
3D culture of human pluripotent stem cells in RGD-alginate hydrogel improves retinal tissue development.
Acta Biomater. 2017 Feb;49:329-343. doi: 10.1016/j.actbio.2016.11.016. Epub 2016 Nov 5.
3
Generation of Functional Human Retinal Ganglion Cells with Target Specificity from Pluripotent Stem Cells by Chemically Defined Recapitulation of Developmental Mechanism.
Stem Cells. 2017 Mar;35(3):572-585. doi: 10.1002/stem.2513. Epub 2016 Oct 26.
4
Successful Transplantation of Retinal Pigment Epithelial Cells from MHC Homozygote iPSCs in MHC-Matched Models.
Stem Cell Reports. 2016 Oct 11;7(4):635-648. doi: 10.1016/j.stemcr.2016.08.010. Epub 2016 Sep 15.
5
Glaucoma: the retina and beyond.
Acta Neuropathol. 2016 Dec;132(6):807-826. doi: 10.1007/s00401-016-1609-2. Epub 2016 Aug 20.
6
Enriched retinal ganglion cells derived from human embryonic stem cells.
Sci Rep. 2016 Aug 10;6:30552. doi: 10.1038/srep30552.
7
cGMP production of patient-specific iPSCs and photoreceptor precursor cells to treat retinal degenerative blindness.
Sci Rep. 2016 Jul 29;6:30742. doi: 10.1038/srep30742.
8
Function of human pluripotent stem cell-derived photoreceptor progenitors in blind mice.
Sci Rep. 2016 Jul 13;6:29784. doi: 10.1038/srep29784.
9
In Pursuit of Authenticity: Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Clinical Applications.
Stem Cells Transl Med. 2016 Nov;5(11):1562-1574. doi: 10.5966/sctm.2016-0037. Epub 2016 Jul 11.
10
Stem Cell-Derived Photoreceptor Transplants Differentially Integrate Into Mouse Models of Cone-Rod Dystrophy.
Invest Ophthalmol Vis Sci. 2016 Jun 1;57(7):3509-20. doi: 10.1167/iovs.16-19087.
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