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[视网膜色素变性的光遗传学治疗研究进展]

[Research progress in optogenetic therapy for retinitis pigmentosa].

作者信息

Liang J, Fang D, Chen L, Diao Y Y, Xie T, Zou Z H, Zheng H Y, Zhang S C

机构信息

Shenzhen Eye Hospital, Jinan University, Shenzhen Institute of Eye Disease Control, Shenzhen518040, China.

出版信息

Zhonghua Yan Ke Za Zhi. 2025 Jan 11;61(1):66-70. doi: 10.3760/cma.j.cn112142-20240321-00128.

Abstract

Retinitis pigmentosa (RP) is a group of inherited retinal diseases characterized by progressive loss of photoreceptor cells and retinal pigment epithelium function. Its treatment has long been a focus and challenge in ophthalmic research. Despite advances in therapies such as stem cell transplantation, gene therapy, and retinal prosthetic implants, many difficulties remain. Optogenetic technology, an emerging biological technique combining optics and genetics, holds promise for treating RP by inducing the expression of light-sensitive proteins in degenerated retinal cells, thereby partially restoring patients' vision and light sensitivity. However, limitations such as difficulties in selecting target cells, potential risks of viral transfection, and uncertainties in visual restoration still exist. With the continuous improvement of light sensitivity of optogenetic proteins and the development of viral vectors with higher transfection efficiency, it is believed that optogenetic therapy will help more patients regain vision.

摘要

视网膜色素变性(RP)是一组遗传性视网膜疾病,其特征是光感受器细胞和视网膜色素上皮功能逐渐丧失。其治疗长期以来一直是眼科研究的重点和挑战。尽管在干细胞移植、基因治疗和视网膜假体植入等治疗方法上取得了进展,但仍存在许多困难。光遗传学技术是一种将光学和遗传学相结合的新兴生物技术,有望通过诱导退化视网膜细胞中光敏蛋白的表达来治疗RP,从而部分恢复患者的视力和光敏感度。然而,仍存在诸如选择靶细胞困难、病毒转染的潜在风险以及视觉恢复的不确定性等局限性。随着光遗传学蛋白光敏感度的不断提高以及转染效率更高的病毒载体的发展,相信光遗传学疗法将帮助更多患者恢复视力。

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