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光遗传学介导的基因治疗视网膜疾病。

Optogenetics-Mediated Gene Therapy for Retinal Diseases.

机构信息

Laboratory of Visual Neuroscience, Department of Chemistry and Biological Sciences, Faculty of Science and Engineering, Iwate University, Morioka, Iwate, Japan.

出版信息

Adv Exp Med Biol. 2021;1293:535-543. doi: 10.1007/978-981-15-8763-4_37.

DOI:10.1007/978-981-15-8763-4_37
PMID:33398840
Abstract

The visual system consists of various types of neurons and a single-nucleotide mutation can sometimes lead to blindness. The phototransduction pathway in the retina starts from the first-order neurons, the photoreceptor cells, and transmits the signals to second-order neurons. Finally, the output signal from third-order neurons, the ganglion cells, is carried to the brain. The photoreceptor cells are the only neurons in the retina that can respond to a light signal; they are hyperpolarised when they receive light, and the ganglion cells carry the signals to the brain following the depolarization. Recently, various types of channelrhodopsins have been found and developed. It is expected that the gene therapies using the cation channel as well as anion channelrhodopsin genes would be effective against diseases that cause severe destruction of visual function, such as the retinitis pigmentosa and age-related macular degeneration. In this review, we mainly describe mVChR1-mediated gene therapy for retinitis pigmentosa and the future application of optogenetic genes in retinal diseases.

摘要

视觉系统由各种类型的神经元组成,单个核苷酸突变有时会导致失明。视网膜中的光转导途径始于一级神经元,即光感受器细胞,并将信号传递给二级神经元。最后,来自三级神经元,即节细胞的输出信号,被传递到大脑。光感受器细胞是视网膜中唯一能对光信号做出反应的神经元;当它们接收到光时会超极化,而节细胞在去极化后将信号传递到大脑。最近,已经发现并开发了各种类型的通道视紫红质。预计使用阳离子通道和阴离子通道视紫红质基因的基因治疗将对导致严重视觉功能丧失的疾病(如色素性视网膜炎和年龄相关性黄斑变性)有效。在这篇综述中,我们主要描述了 mVChR1 介导的色素性视网膜炎基因治疗以及光遗传学基因在视网膜疾病中的未来应用。

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-Methyl--Nitrosourea-Induced Photoreceptor Degeneration Is Inhibited by Nicotinamide via the Blockade of Upstream Events before the Phosphorylation of Signalling Proteins.烟酰胺通过阻止信号蛋白磷酸化上游事件抑制甲基亚硝脲诱导的光感受器变性。
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