雑誌文献を検索します。書籍を検索する際には「書籍検索」を選択してください。

検索

書誌情報 詳細検索 by 医中誌

Japanese

Current Status of Gene Therapy for Parkinson Disease Hideki Mochizuki 1 1Department of Neurology,Research Institute for Diseases of Old Ages Juntendo University Keyword: Parkinson disease , gene therapy , AAV , parkin pp.485-493
Published Date 2009/4/1
DOI https://doi.org/10.11477/mf.1416100473
  • Abstract
  • Look Inside
  • Reference

Abstract

 Gene therapy is particularly appropriate for Parkinson disease (PD) since this condition exclusively affects the dopaminergic neurons projecting from the substantia nigra pars compacta (SNc) to the putamen. Currently,4 ongoing phase I clinical trials are utilizing recombinant adeno-associated viral vectors (rAAv) or lentivirus vectors for the treatment of PD. In this article,we describe recent progress in the development of gene therapy methods for PD by reviewing clinical trials in this field. Parkin-associated PD is recessively inherited,that is,loss of function of parkin leads to the development of parkin -associated PD; hence,substrates for parkin (for its E3 function) are expected to accumulate in the brain. Therefore,the replacement of parkin function in such patients would decrease the toxicity of these substrates. We previously found that the transfer of parkin,encoding a familial PD-linked E3 ubiquitin ligase,in rats with PD could prevent the degeneration of nigral dopaminergic neurons. In addition,we recently reported the case report of a preclinical examination of rAAV vector-mediated retrograde delivery of parkin into nigrostriatal dopaminergic neurons in a non-human primate. In this article,we also review the potential of parkin gene therapy for the treatment of PD patients.


Copyright © 2009, Igaku-Shoin Ltd. All rights reserved.

基本情報

電子版ISSN 1344-8129 印刷版ISSN 1881-6096 医学書院

関連文献

もっと見る

文献を共有