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光遗传学方法在色素性视网膜炎中的视觉功能恢复。

Optogenetic approaches to restoring visual function in retinitis pigmentosa.

机构信息

Neural Circuit Laboratories, Friedrich Miescher Institute for Biomedical Research, Basel, Switzerland.

出版信息

Curr Opin Neurobiol. 2011 Dec;21(6):942-6. doi: 10.1016/j.conb.2011.06.001. Epub 2011 Jun 25.

DOI:10.1016/j.conb.2011.06.001
PMID:21708457
Abstract

Retinitis pigmentosa is a hereditary eye disease that affects photoreceptors and leads to blindness. The discovery of a microbial light-gated channel and the subsequent development of similar 'optogenetic' sensors have opened the door to creating artificial photoreceptors in the remaining retinal circuits of retinitis pigmentosa retinas via gene therapy. Here we review recent studies in animal models of retinitis pigmentosa that have combined knowledge of retinal cell types, circuits and computations with the ability to equip cell types with optogenetic sensors in order to restore visual activity. We also discuss the translational potential of this therapy.

摘要

色素性视网膜炎是一种遗传性眼病,会影响光感受器,导致失明。微生物光门控通道的发现,以及类似的“光遗传学”传感器的后续开发,为通过基因治疗在色素性视网膜炎视网膜的剩余视网膜电路中创建人工光感受器打开了大门。在这里,我们回顾了色素性视网膜炎动物模型的最新研究,这些研究结合了视网膜细胞类型、回路和计算的知识,以及为细胞类型配备光遗传学传感器以恢复视觉活动的能力。我们还讨论了这种治疗方法的转化潜力。

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1
Optogenetic approaches to restoring visual function in retinitis pigmentosa.光遗传学方法在色素性视网膜炎中的视觉功能恢复。
Curr Opin Neurobiol. 2011 Dec;21(6):942-6. doi: 10.1016/j.conb.2011.06.001. Epub 2011 Jun 25.
2
Optogenetic therapy for retinitis pigmentosa.光遗传学疗法治疗色素性视网膜炎。
Gene Ther. 2012 Feb;19(2):169-75. doi: 10.1038/gt.2011.155. Epub 2011 Oct 13.
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[Retinitis pigmentosa: eye sight restoration by optogenetic therapy].[视网膜色素变性:通过光遗传学疗法恢复视力]
Biol Aujourdhui. 2013;207(2):109-21. doi: 10.1051/jbio/2013011. Epub 2013 Oct 10.
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Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa.遗传性激活视锥细胞可恢复色素性视网膜炎的视觉反应。
Science. 2010 Jul 23;329(5990):413-7. doi: 10.1126/science.1190897. Epub 2010 Jun 24.
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[Therapeutic strategies in RP (retinitis pigmentosa): light at the end of the tunnel?].[视网膜色素变性的治疗策略:曙光在望?]
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Gene therapy for retinitis pigmentosa.视网膜色素变性的基因治疗。
Curr Opin Mol Ther. 2000 Aug;2(4):420-5.
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RPGR and RP2: targets for the treatment of X-linked retinitis pigmentosa?RPGR和RP2:治疗X连锁视网膜色素变性的靶点?
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Prospects for retinal gene replacement therapy.视网膜基因替代疗法的前景。
Trends Genet. 2009 Apr;25(4):156-65. doi: 10.1016/j.tig.2009.02.003. Epub 2009 Mar 18.
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[Recent progress in molecular genetics and gene therapy for retinitis pigmentosa].
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