期刊
GENE THERAPY
卷 13, 期 6, 页码 525-531出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.gt.3302664
关键词
Huntington's disease; spinocerebellar ataxia; adeno-associated virus vectors; RNAi; polyglutamine
类别
资金
- NICHD NIH HHS [HD44093] Funding Source: Medline
- NINDS NIH HHS [NS050210] Funding Source: Medline
Genetic diseases that are accompanied by central nervous system involvement are often fatal. Among these are the autosomal dominant neurogenetic diseases caused by nucleotide repeat expansion. For example, Huntington's disease (HD) and spinal cerebellar ataxia are caused by expansion of a tract of CAGs encoding glutamine. In HD and the other CAG-repeat expansion diseases, the expansion is in the coding region. Myotonic dystrophy is caused by repeat expansions of CUG or CCTG in noncoding regions, and the mutant RNA is disease causing. Treatments for these disorders are limited to symptomatic intervention. RNA interference (RNAi), which is a method for inhibiting target gene expression, provides a unique tool for therapy by attacking the fundamental problem directly. In this review, we describe briefly several representative disorders and their respective molecular targets, and methods to accomplish therapeutic RNAi. Finally, we summarize studies performed to date.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据