Allele-specific RNA interference for neurological disease

被引：70
|
作者：
Rodriguez-Lebron, E [1 ]
Paulson, HL [1 ]
机构：
[1] Carver Coll Med, Dept Neurol, Iowa City, IA USA
来源：
GENE THERAPY | 2006年 / 13卷 / 06期
关键词：
siRNA; miRNA; allele-specific; dominant; neurodegenerative; delivery;
D O I：
10.1038/sj.gt.3302702
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
Suppressing the expression of toxic genes through RNAi holds great promise for the treatment of human disease. Allele-specific approaches have now been used to silence dominant toxic genes implicated in several neurological disorders. Here, we review strategies used to achieve allele-specific silencing in light of recent developments in the field of RNAi biology. In particular, new insights into siRNA and miRNA processing may be used to improve efficiency and specificity of RNAi therapy. We further discuss steps that can be taken to maximize the therapeutic benefits of this powerful technology.
引用
收藏
页码：576 / 581
页数：6
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