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神经退行性疾病的基因治疗:进展、见解与前景

Gene therapy for neurodegenerative disorders: advances, insights and prospects.

作者信息

Chen Wei, Hu Yang, Ju Dianwen

机构信息

Department of Biological Medicines, Fudan University School of Pharmacy, Shanghai 201203, China.

Department of Ophthalmology, Stanford University School of Medicine, Palo Alto, CA 94304, USA.

出版信息

Acta Pharm Sin B. 2020 Aug;10(8):1347-1359. doi: 10.1016/j.apsb.2020.01.015. Epub 2020 Jan 31.

Abstract

Gene therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of neurodegenerative disorders, including Alzheimer's disease (AD), Parkinson's disease (PD) and Huntington's disease (HD). Some early clinical trials have failed to achieve satisfactory therapeutic effects. Efforts to enhance effectiveness are now concentrating on three major fields: identification of new vectors, novel therapeutic targets, and reliable of delivery routes for transgenes. These approaches are being assessed closely in preclinical and clinical trials, which may ultimately provide powerful treatments for patients. Here, we discuss advances and challenges of gene therapy for neurodegenerative disorders, highlighting promising technologies, targets, and future prospects.

摘要

基因治疗正迅速成为一种针对多种神经退行性疾病的强大治疗策略,这些疾病包括阿尔茨海默病(AD)、帕金森病(PD)和亨廷顿舞蹈病(HD)。一些早期临床试验未能取得令人满意的治疗效果。目前提高疗效的努力主要集中在三个主要领域:新型载体的鉴定、新的治疗靶点以及转基因递送途径的可靠性。这些方法正在临床前和临床试验中得到密切评估,最终可能为患者提供有效的治疗方法。在此,我们讨论神经退行性疾病基因治疗的进展与挑战,重点介绍有前景的技术、靶点和未来前景。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/27db/7488363/6d00a1bb89ff/fx1.jpg

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