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[帕金森病的基因治疗研究]

[Studies of gene therapy for Parkinson's disease].

作者信息

Mochizuki Hideki

机构信息

Department of Neurology, Kitasato University.

出版信息

Rinsho Shinkeigaku. 2009 Nov;49(11):753-5. doi: 10.5692/clinicalneurol.49.753.

Abstract

Currently, four Phase I clinical trials are underway utilizing recombinant adeno-associated viral (rAAV) vectors for the treatment of Parkinson's disease. The vehicle used mainly for gene delivery to the human brain is rAAV vector, which is non-pathogenic and non-self-amplifying. At present, the gene therapy approach is not the best way for the treatment of PD patients, but we believe that the further progress is anticipated toward making this strategy a therapeutic option for PD in the future. This article will review currently ongoing clinical trials of PD gene therapy and then introduce our studies about the gene therapy for PD.

摘要

目前,有四项利用重组腺相关病毒(rAAV)载体治疗帕金森病的I期临床试验正在进行。主要用于将基因递送至人脑的载体是rAAV载体,它无致病性且不会自我扩增。目前,基因治疗方法并非治疗帕金森病患者的最佳方式,但我们相信未来有望取得进一步进展,使该策略成为帕金森病的一种治疗选择。本文将综述目前正在进行的帕金森病基因治疗临床试验,然后介绍我们关于帕金森病基因治疗的研究。

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