mRNA沉默中的靶标选择性

Target selectivity in mRNA silencing.

作者信息

Aronin N

机构信息

University of Massachusetts, Worcester, MA 1655, USA.

出版信息

Gene Ther. 2006 Mar;13(6):509-16. doi: 10.1038/sj.gt.3302726.

DOI:10.1038/sj.gt.3302726

PMID:16520821

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7091803/

Abstract

Despite the excitement and promise of RNA interference in treating neurodegenerative disease, disease gene mRNA might resist mRNA silencing. Conventional siRNA design does not uniformly distinguish a mutant from a wild-type allele. CAG expansions in trinucleotide repeat diseases are unselective targets for small siRNAs. This review will consider recent discoveries in mechanisms of RNA interference and siRNA modifications that improve siRNA selectivity, delivery and performance.

摘要

尽管RNA干扰在治疗神经退行性疾病方面令人兴奋且前景广阔，但疾病基因的信使核糖核酸（mRNA）可能会抵抗mRNA沉默。传统的小干扰RNA（siRNA）设计并不能始终如一地区分突变等位基因和野生型等位基因。三核苷酸重复疾病中的CAG重复扩增是小siRNA的非选择性靶点。本综述将探讨RNA干扰机制和siRNA修饰方面的最新发现，这些发现可提高siRNA的选择性、递送效率和性能。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8f85/7091803/28737c696abd/41434_2006_Article_BF3302726_Fig1_HTML.jpg

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mRNA沉默中的靶标选择性

Target selectivity in mRNA silencing.

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mRNA沉默中的靶标选择性

Target selectivity in mRNA silencing.

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机构信息

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