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Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway

Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway A paper in Nature last year appeared to spell trouble for the prospects of RNA interference-mediated silencing as gene therapy. It showed that large doses of short hairpin RNA disrupted the microRNA pathway in mice, with fatal results. Now a new study suggests that it is too soon to write off RNA therapy. A different type of inhibitory RNA, small interfering RNAs (siRNAs), can be administered to mice without toxicity. The activity of liver microRNAs remains unaffected by siRNAs, despite 80% silencing of target genes in mouse and hamster liver cells. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Nature Springer Journals

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References (12)

Publisher
Springer Journals
Copyright
Copyright © 2007 by Nature Publishing Group
Subject
Science, Humanities and Social Sciences, multidisciplinary; Science, Humanities and Social Sciences, multidisciplinary; Science, multidisciplinary
ISSN
0028-0836
eISSN
1476-4687
DOI
10.1038/nature06179
Publisher site
See Article on Publisher Site

Abstract

A paper in Nature last year appeared to spell trouble for the prospects of RNA interference-mediated silencing as gene therapy. It showed that large doses of short hairpin RNA disrupted the microRNA pathway in mice, with fatal results. Now a new study suggests that it is too soon to write off RNA therapy. A different type of inhibitory RNA, small interfering RNAs (siRNAs), can be administered to mice without toxicity. The activity of liver microRNAs remains unaffected by siRNAs, despite 80% silencing of target genes in mouse and hamster liver cells.

Journal

NatureSpringer Journals

Published: Sep 26, 2007

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