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 and David Bumcrot ()
Additional contact information
Matthias John: Alnylam Europe AG, Fritz-Hornschuch-Str. 9, 95326 Kulmbach, Germany
Rainer Constien: Alnylam Europe AG, Fritz-Hornschuch-Str. 9, 95326 Kulmbach, Germany
Akin Akinc: Alnylam Pharmaceuticals Inc., 300 Third Street, Cambridge, Massachusetts 02142, USA
Michael Goldberg: and
Young-Ah Moon: University of Texas Southwestern Medical Center, Dallas, Texas 75390-9046, USA
Martina Spranger: Institute of Molecular Systems Biology, Swiss Federal Institute of Technology ETH Zürich, HPT E73, CH-8093 Zürich, Switzerland
Philipp Hadwiger: Alnylam Europe AG, Fritz-Hornschuch-Str. 9, 95326 Kulmbach, Germany
Jürgen Soutschek: Alnylam Europe AG, Fritz-Hornschuch-Str. 9, 95326 Kulmbach, Germany
Hans-Peter Vornlocher: Alnylam Europe AG, Fritz-Hornschuch-Str. 9, 95326 Kulmbach, Germany
Muthiah Manoharan: Alnylam Pharmaceuticals Inc., 300 Third Street, Cambridge, Massachusetts 02142, USA
Markus Stoffel: Institute of Molecular Systems Biology, Swiss Federal Institute of Technology ETH Zürich, HPT E73, CH-8093 Zürich, Switzerland
Robert Langer: and
Daniel G. Anderson: Center for Cancer Research, Massachusetts Institute of Technology, 77 Massachusetts Avenue, Cambridge, Massachusetss 02139, USA
Jay D. Horton: University of Texas Southwestern Medical Center, Dallas, Texas 75390-9046, USA
Victor Koteliansky: Alnylam Pharmaceuticals Inc., 300 Third Street, Cambridge, Massachusetts 02142, USA
David Bumcrot: Alnylam Pharmaceuticals Inc., 300 Third Street, Cambridge, Massachusetts 02142, USA

Nature, 2007, vol. 449, issue 7163, 745-747

Abstract: 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.

Date: 2007
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DOI: 10.1038/nature06179

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