Advances in Ophthalmic Optogenetics: Approaches and Applications.
Advances in Ophthalmic Optogenetics: Approaches and Applications.
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DOI：
10.3390/biom12020269
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发表时间：
2022-02-08
期刊：
影响因子：
5.5
通讯作者：
Sun Y
中科院分区：
文献类型：
--
作者：
Prosseda PP;Tran M;Kowal T;Wang B;Sun Y
光遗传学的最新进展为退行性眼病的视力恢复带来了希望。光遗传学是指利用光来控制靶细胞的细胞活性的技术。虽然光遗传学是一项相对较新的技术，但在临床前和I/II期临床试验中已经探索了多种治疗选择，旨在为主要致盲性眼病（如青光眼和视网膜色素变性）开发新型，安全和有效的治疗方法。视觉恢复的光遗传学方法主要旨在通过将光敏蛋白插入到没有内在光敏性的下游视网膜神经元中来替换丢失或功能障碍的光感受器。这种方法是有吸引力的，因为它们对视网膜变性的遗传原因是不可知的，这带来了希望，所有形式的视网膜营养不良和退行性疾病都可以治疗。光遗传学策略也可以通过作为研究视网膜变性发病机制和确定临床相关治疗靶点的重要工具对转化研究产生深远影响。例如，最近已经将OCR-CIBN光遗传学系统应用于青光眼的动物模型，表明OCRL在小梁网中的眼内压调节中的潜在作用。随着光遗传学策略的深入研究，考虑这种疗法可能提供的机遇和挑战似乎至关重要。在此，我们回顾了最近有希望的光遗传学分子，载体，以及光遗传学在视网膜变性和青光眼治疗中的应用。我们还总结了正在进行的视力恢复临床试验的初步结果。
Recent advances in optogenetics hold promise for vision restoration in degenerative eye diseases. Optogenetics refers to techniques that use light to control the cellular activity of targeted cells. Although optogenetics is a relatively new technology, multiple therapeutic options are already being explored in pre-clinical and phase I/II clinical trials with the aim of developing novel, safe, and effective treatments for major blinding eye diseases, such as glaucoma and retinitis pigmentosa. Optogenetic approaches to visual restoration are primarily aimed at replacing lost or dysfunctional photoreceptors by inserting light-sensitive proteins into downstream retinal neurons that have no intrinsic light sensitivity. Such approaches are attractive because they are agnostic to the genetic causes of retinal degeneration, which raises hopes that all forms of retinal dystrophic and degenerative diseases could become treatable. Optogenetic strategies can also have a far-reaching impact on translational research by serving as important tools to study the pathogenesis of retinal degeneration and to identify clinically relevant therapeutic targets. For example, the CRY-CIBN optogenetic system has been recently applied to animal models of glaucoma, suggesting a potential role of OCRL in the regulation of intraocular pressure in trabecular meshwork. As optogenetic strategies are being intensely investigated, it appears crucial to consider the opportunities and challenges such therapies may offer. Here, we review the more recent promising optogenetic molecules, vectors, and applications of optogenetics for the treatment of retinal degeneration and glaucoma. We also summarize the preliminary results of ongoing clinical trials for visual restoration.
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