眼科光遗传学进展：方法和应用,Biomolecules

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眼科光遗传学进展：方法和应用
Biomolecules ( IF 4.8 ) Pub Date : 2022-02-09 , DOI: 10.3390/biom12020269
Philipp P Prosseda ₁ , Matthew Tran _{1,

2} , Tia Kowal _{1,

3} , Biao Wang ₁ , Yang Sun _{1,

3}

Affiliation

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。光遗传学是指使用光来控制目标细胞的细胞活性的技术。尽管光遗传学是一项相对较新的技术，但临床前和 I/II 期临床试验已经在探索多种治疗方案，目的是为青光眼和色素性视网膜炎等主要致盲性眼病开发新颖、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没有内在光敏性的下游视网膜神经元来替代丢失或功能失调的光感受器。这种方法很有吸引力，因为它们不知道视网膜退化的遗传原因，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愈。光遗传学策略还可以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，CRY-CIBN 光遗传学系统最近已应用于青光眼动物模型，表明 OCRL 在调节小梁网眼压中具有潜在作用。随着光遗传学策略的深入研究，考虑此类疗法可能带来的机遇和挑战似乎至关重要。在这里，我们回顾了最近有前景的光遗传学分子、载体和光遗传学在视网膜变性和青光眼治疗中的应用。

"点击查看英文标题和摘要"

Advances in Ophthalmic Optogenetics: Approaches and Applications

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.

更新日期：2022-02-09

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