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用化学光开关使盲人重见光明。

Restoring Vision to the Blind with Chemical Photoswitches.

机构信息

F.M. Kirby Neurobiology Center , Boston Children's Hospital , Boston , Massachusetts 02115 , United States.

Department of Neurobiology , Harvard Medical School , Boston , Massachusetts 02115 , United States.

出版信息

Chem Rev. 2018 Nov 14;118(21):10748-10773. doi: 10.1021/acs.chemrev.7b00723. Epub 2018 Jun 6.

DOI:10.1021/acs.chemrev.7b00723
PMID:29874052
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6389275/
Abstract

Degenerative retinal diseases such as retinitis pigmentosa (RP) and age-related macular degeneration (AMD) affect millions of people around the world and lead to irreversible vision loss if left untreated. A number of therapeutic strategies have been developed over the years to treat these diseases or restore vision to already blind patients. In this Review, we describe the development and translational application of light-sensitive chemical photoswitches to restore visual function to the blind retina and compare the translational potential of photoswitches with other vision-restoring therapies. This therapeutic strategy is enabled by an efficient fusion of chemical synthesis, chemical biology, and molecular biology and is broadly applicable to other biological systems. We hope this Review will be of interest to chemists as well as neuroscientists and clinicians.

摘要

退行性视网膜疾病,如色素性视网膜炎(RP)和年龄相关性黄斑变性(AMD),影响着全球数以百万计的人,如果不加以治疗,会导致不可逆转的视力丧失。多年来,已经开发出许多治疗这些疾病或恢复已失明患者视力的治疗策略。在这篇综述中,我们描述了光敏化学光开关的发展和转化应用,以恢复盲视网膜的视觉功能,并比较了光开关与其他恢复视力的治疗方法的转化潜力。这种治疗策略是通过化学合成、化学生物学和分子生物学的有效融合实现的,并且广泛适用于其他生物系统。我们希望这篇综述不仅对化学家,而且对神经科学家和临床医生都有兴趣。

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本文引用的文献

1
Voretigene neparvovec-rzyl (Luxturna) for inherited retinal dystrophy.用于遗传性视网膜营养不良的voretigene neparvovec-rzyl(Luxturna)。
Med Lett Drugs Ther. 2018 Mar 26;60(1543):53-55.
2
Restoration of patterned vision with an engineered photoactivatable G protein-coupled receptor.利用工程化的光激活 G 蛋白偶联受体恢复模式化视觉。
Nat Commun. 2017 Nov 30;8(1):1862. doi: 10.1038/s41467-017-01990-7.
3
The Progression of Geographic Atrophy Secondary to Age-Related Macular Degeneration.年龄相关性黄斑变性继发的地图样萎缩进展。
Ophthalmology. 2018 Mar;125(3):369-390. doi: 10.1016/j.ophtha.2017.08.038. Epub 2017 Oct 27.
4
Gene therapy for inherited retinal and optic nerve degenerations.遗传性视网膜和视神经退行性疾病的基因治疗。
Expert Opin Biol Ther. 2018 Jan;18(1):37-49. doi: 10.1080/14712598.2018.1389886. Epub 2017 Oct 23.
5
Specificity and Speed: Tethered Photopharmacology.特异性与速度:系留光药理学
Biochemistry. 2017 Oct 3;56(39):5214-5220. doi: 10.1021/acs.biochem.7b00687. Epub 2017 Sep 14.
6
Recent developments in age-related macular degeneration: a review.年龄相关性黄斑变性的最新进展:综述
Clin Interv Aging. 2017 Aug 22;12:1313-1330. doi: 10.2147/CIA.S143508. eCollection 2017.
7
Precise modulation of neuronal activity with synthetic photoswitchable ligands.用合成光开关配体精确调节神经元活动。
Curr Opin Neurobiol. 2017 Aug;45:202-209. doi: 10.1016/j.conb.2017.05.021. Epub 2017 Jul 6.
8
Understanding and improving photo-control of ion channels in nociceptors with azobenzene photo-switches.理解并改善用偶氮苯光开关控制伤害感受器中离子通道的光控作用。
Br J Pharmacol. 2018 Jun;175(12):2296-2311. doi: 10.1111/bph.13923. Epub 2017 Jul 27.
9
Photopharmacological control of bipolar cells restores visual function in blind mice.双极细胞的光药理学控制恢复了盲鼠的视觉功能。
J Clin Invest. 2017 Jun 30;127(7):2598-2611. doi: 10.1172/JCI92156. Epub 2017 Jun 5.
10
Progress of stem/progenitor cell-based therapy for retinal degeneration.基于干细胞/祖细胞的视网膜变性治疗进展
J Transl Med. 2017 May 10;15(1):99. doi: 10.1186/s12967-017-1183-y.