视网膜退行性疾病的光基因治疗综述。

IF 1.4 4区 医学 Q3 OPHTHALMOLOGY
Ehab Kasem, Masa Watfa, Ali Afif, Raghad Hasan, Majd Mansour, Haya Almhmoud, Basem Zaino
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引用次数: 0

摘要

光遗传学是一种新型基因操作和药物发现的前沿工具,对包括视网膜疾病在内的各种神经系统疾病具有重要的治疗潜力。视网膜疾病,如年龄相关性黄斑变性(AMD)和视网膜色素变性(RP),由于治疗选择有限和普遍缺乏认识,严重损害生活质量并导致严重的视力障碍。这些退行性疾病的发病率越来越高,因此需要创新的解决方案,如光遗传学。光遗传疗法引入编码光敏蛋白的基因,这些蛋白受光信号控制,使神经元具有光敏性。这种精确的靶向方法不需要特定的基因干预,可以绕过功能失调的光感受器,为各种退行性和营养不良眼病提供了一种治疗选择。晚期遗传性视网膜疾病患者的成功结果和大量临床试验表明,光遗传学可能是一种有效的人类治疗方法。本文综述了光基因治疗的现状,讨论了其面临的挑战,并总结了正在进行的神经和视觉恢复临床试验的结果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Optogenetic therapy for retinal degenerative diseases: A review.

Optogenetics, a cutting-edge tool in novel gene manipulation and drug discovery, holds significant therapeutic potential for a variety of neurological disorders, including retinal diseases. Retinal diseases such as age-related macular degeneration (AMD) and retinitis pigmentosa (RP), significantly impair quality of life and cause severe visual impairment due to limited treatment options and a general lack of awareness. The increasing incidence of these degenerative conditions underscores the need for innovative solutions, such as optogenetics. Optogenetic therapy introduces genes coding for light-sensitive proteins, which are controlled by light signals to make neurons photosensitive. This precise targeting approach does not require specific gene intervention and can bypass dysfunctional photoreceptors, offering a treatment option for various degenerative and dystrophic eye diseases. Successful outcomes in patients with late-stage genetic retinal diseases and numerous clinical trials suggest that optogenetics could be an effective treatment for humans. This review provides an overview of the current landscape of optogenetic therapy, discusses its challenges, and summarizes the findings of ongoing clinical trials for neural and visual restoration.

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来源期刊
CiteScore
3.60
自引率
0.00%
发文量
372
审稿时长
3-8 weeks
期刊介绍: The European Journal of Ophthalmology was founded in 1991 and is issued in print bi-monthly. It publishes only peer-reviewed original research reporting clinical observations and laboratory investigations with clinical relevance focusing on new diagnostic and surgical techniques, instrument and therapy updates, results of clinical trials and research findings.
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