Editor's Summary

11 October 2007

RNAi therapy back on track

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.

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

Matthias John, Rainer Constien, Akin Akinc, Michael Goldberg, Young-Ah Moon, Martina Spranger, Philipp Hadwiger, Jürgen Soutschek, Hans-Peter Vornlocher, Muthiah Manoharan, Markus Stoffel, Robert Langer, Daniel G. Anderson, Jay D. Horton, Victor Koteliansky & David Bumcrot

doi:10.1038/nature06179

First paragraph | Full Text | PDF (289K) | Supplementary information