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视网膜色素变性与治疗候选药物

Retinitis Pigmentosa and Therapeutic Candidates.

作者信息

Livingston Chelsea A, Weng Christina Y, Chancellor John R

机构信息

Baylor College of Medicine, Cullen Eye Institute, Houston, TX.

出版信息

Int Ophthalmol Clin. 2025 Jan 1;65(1):17-21. doi: 10.1097/IIO.0000000000000542. Epub 2024 Dec 23.

Abstract

Retinitis pigmentosa (RP) is a class of inherited retinal dystrophies (IRDs) that involves the degeneration of retinal photoreceptor cells and results in progressive vision loss. It was identified and named in 1857. For over 100 years, treatment of RP was generally limited to modifications in diet, management of cystoid macular edema, and supportive care for low vision. Over the last several decades, advances in technology and our understanding of the human genome have led to a host of new therapeutic candidates for the treatment of RP. This includes gene and cell therapy, optogenetics, neuroprotective agents, and electronic retinal implants. In this article, we summarize both the traditional and novel therapeutic modalities for the treatment of retinitis pigmentosa.

摘要

视网膜色素变性(RP)是一类遗传性视网膜营养不良(IRD),涉及视网膜光感受器细胞的退化,并导致进行性视力丧失。它于1857年被发现并命名。在过去的100多年里,RP的治疗通常仅限于饮食调整、黄斑囊样水肿的管理以及对低视力的支持性护理。在过去几十年中,技术的进步以及我们对人类基因组的了解催生了许多治疗RP的新候选疗法。这包括基因和细胞疗法、光遗传学、神经保护剂以及电子视网膜植入物。在本文中,我们总结了治疗视网膜色素变性的传统和新型治疗方法。

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