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

Author

Listed:
  • 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
    Center for Cancer Research, Massachusetts Institute of Technology, 77 Massachusetts Avenue, Cambridge, Massachusetss 02139, USA)

  • 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)

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.

Suggested Citation

  • Matthias John & Rainer Constien & Akin Akinc & Michael Goldberg & Young-Ah Moon & Martina Spranger & Philipp Hadwiger & Jürgen Soutschek & Hans-Peter Vornlocher & Muthiah Manoharan & Markus Stoffel & , 2007. "Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway," Nature, Nature, vol. 449(7163), pages 745-747, October.
    • Handle: RePEc:nat:nature:v:449:y:2007:i:7163:d:10.1038_nature06179
    DOI: 10.1038/nature06179
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