RNAi medicine for the brain: progresses and challenges

被引：53

作者：

Boudreau, Ryan L. ^{[1
]}

Rodriguez-Lebron, Edgardo ^{[1
]}

Davidson, Beverly L. ^{[1
,2
,3
]}

机构：

[1] Univ Iowa, Dept Internal Med, Iowa City, IA 52242 USA

[2] Univ Iowa, Dept Neurol, Iowa City, IA 52242 USA

[3] Univ Iowa, Dept Mol Physiol & Biophys, Iowa City, IA 52242 USA

来源：

HUMAN MOLECULAR GENETICS | 2011年 / 20卷

基金：

美国国家卫生研究院;

关键词：

SMALL INTERFERING RNA; HUNTINGTONS-DISEASE; ALPHA-SYNUCLEIN; ALZHEIMERS-DISEASE; IN-VITRO; MUTANT HUNTINGTIN; PASSENGER-STRAND; MESSENGER-RNAS; RAT MODEL; SIRNA;

D O I：

10.1093/hmg/ddr137

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

070307 [化学生物学]; 071010 [生物化学与分子生物学];

摘要：

RNAi interference (RNAi) is a powerful gene silencing technology that has immense potential for treating a vast array of human ailments, for which suppressing disease-associated genes may provide clinical benefit. Here, we review the development of RNAi as a therapeutic modality for neurodegenerative diseases affecting the central nervous system (CNS). We overview promising preclinical data for the application of RNAi in the CNS and discuss key challenges (e. g. delivery and specificity) that remain as these approaches transition to the clinic.

引用

页码：R21 / R27

页数：7

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