Department of Biotechnology, Chemistry & Pharmacy, Department of Excellence 2018-2022, University of Siena, Via A. Moro 2, 53100 Siena, Italy.
Department of Pharmacy, Department of Excellence 2018-2022, University of Naples Federico II, Via D. Montesano 49, 80131 Napoli, Italy.
Future Med Chem. 2019 Mar;11(5):463-487. doi: 10.4155/fmc-2018-0315. Epub 2019 Mar 25.
In the last decade, innovative therapeutic strategies against inherited retinal degenerations (IRDs) have emerged. In particular, chemical- and opto-genetics approaches or a combination of them have been identified for modulating neuronal/optical activity in order to restore vision in blinding diseases. The 'chemical-genetics approach' (optopharmacology) uses small molecules (exogenous photoswitches) for restoring light sensitivity by activating ion channels. The 'opto-genetics approach' employs light-activated photosensitive proteins (exogenous opsins), introduced by viral vectors in injured tissues, to restore light response. These approaches offer control of neuronal activities with spatial precision and limited invasiveness, although with some drawbacks. Currently, a combined therapeutic strategy (optogenetic pharmacology) is emerging. This review describes the state of the art and provides an overview of the future perspectives in vision restoration.
在过去的十年中，出现了针对遗传性视网膜退行性疾病 (IRDs) 的创新治疗策略。特别是，已经确定了化学和光遗传学方法或它们的组合，用于调节神经元/光学活性，以恢复致盲疾病的视力。“化学遗传学方法”（光药理学）使用小分子（外源性光开关）通过激活离子通道来恢复光敏感性。“光遗传学方法”采用光激活光敏蛋白（外源性视蛋白），通过病毒载体引入受损组织，以恢复光反应。这些方法提供了具有空间精度和有限侵入性的神经元活动控制，尽管存在一些缺点。目前，一种联合治疗策略（光遗传学药理学）正在出现。本文综述了该领域的最新技术，并概述了视力恢复的未来前景。
