Gene therapy for neurodegenerative disorders:advances, insights and prospects

作     者：Wei Chen Yang Hu Dianwen Ju Wei Chen;Yang Hu;Dianwen Ju

作者机构：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 物：《Acta Pharmaceutica Sinica B》 (药学学报（英文版）)

年 卷 期：2020年第10卷第8期

页      面：1347-1359页

核心收录：

学科分类：1002[医学-临床医学] 100204[医学-神经病学] 10[医学] 

基　　金：supported by National Natural Science Foundation of China(Nos.81773620 and 81573332) National Key Basic Research Program of China(No.2015CB931800)to Dianwen Ju NIH NEI(EY024932,EY023295 and EY028106,USA)to Yang Hu 

主　　题：disorders degenerative Huntington 

摘      要：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.