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Gene-independent therapeutic interventions to maintain and restore light sensitivity in degenerating photoreceptors
Progress in Retinal and Eye Research ( IF 17.8 ) Pub Date : 2022-05-11 , DOI: 10.1016/j.preteyeres.2022.101065
Marta Zuzic 1 , Johannes Striebel 1 , Julia S Pawlick 1 , Kritika Sharma 1 , Frank G Holz 1 , Volker Busskamp 1
Affiliation  

Neurodegenerative retinal diseases are a prime cause of blindness in industrialized countries. In many cases, there are no therapeutic treatments, although they are essential to improve patients' quality of life. A set of disease-causing genes, which primarily affect photoreceptors, has already been identified and is of major interest for developing gene therapies. Nevertheless, depending on the nature and the state of the disease, gene-independent strategies are needed. Various strategies to halt disease progression or maintain function of the retina are under research. These therapeutic interventions include neuroprotection, direct reprogramming of affected photoreceptors, the application of non-coding RNAs, the generation of artificial photoreceptors by optogenetics and cell replacement strategies. During recent years, major breakthroughs have been made such as the first optogenetic application to a blind patient whose visual function partially recovered by targeting retinal ganglion cells. Also, RPE cell transplantation therapies are under clinical investigation and show great promise to improve visual function in blind patients. These cells are generated from human stem cells. Similar therapies for replacing photoreceptors are extensively tested in pre-clinical models. This marks just the start of promising new cures taking advantage of developments in the areas of genetic engineering, optogenetics, and stem-cell research. In this review, we present the recent therapeutic advances of gene-independent approaches that are currently under clinical evaluation. Our main focus is on photoreceptors as these sensory cells are highly vulnerable to degenerative diseases, and are crucial for light detection.



中文翻译:

维持和恢复退化光感受器光敏感性的基因独立治疗干预

神经退行性视网膜疾病是工业化国家失明的主要原因。在许多情况下,没有治疗方法,尽管它们对于提高患者的生活质量至关重要。一组主要影响光感受器的致病基因已经被确定,并且对于开发基因疗法具有重要意义。然而,根据疾病的性质和状态,需要不依赖基因的策略。正在研究阻止疾病进展或维持视网膜功能的各种策略。这些治疗干预包括神经保护、受影响光感受器的直接重编程、非编码 RNA 的应用、通过光遗传学和细胞替代策略生成人工光感受器。近年来,已经取得了重大突破,例如首次将光遗传学应用于盲人患者,其视觉功能通过靶向视网膜神经节细胞而部分恢复。此外,RPE 细胞移植疗法正在临床研究中,并显示出改善盲人视觉功能的巨大希望。这些细胞是由人类干细胞产生的。用于替代光感受器的类似疗法在临床前模型中进行了广泛测试。这标志着利用基因工程、光遗传学和干细胞研究领域发展的有希望的新疗法的开始。在这篇综述中,我们介绍了目前正在临床评估的基因独立方法的最新治疗进展。

更新日期:2022-05-11
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