RNAi-mediated gene silencing in non-human primates

Tracy S. Zimmermann (), Amy C. H. Lee, Akin Akinc, Birgit Bramlage, David Bumcrot, Matthew N. Fedoruk, Jens Harborth, James A. Heyes, Lloyd B. Jeffs, Matthias John, Adam D. Judge, Kieu Lam, Kevin McClintock, Lubomir V. Nechev, Lorne R. Palmer, Timothy Racie, Ingo Röhl, Stephan Seiffert, Sumi Shanmugam, Vandana Sood, Jürgen Soutschek, Ivanka Toudjarska, Amanda J. Wheat, Ed Yaworski, William Zedalis, Victor Koteliansky, Muthiah Manoharan, Hans-Peter Vornlocher and Ian MacLachlan ()
Additional contact information
Tracy S. Zimmermann: Alnylam Pharmaceuticals Inc.
Amy C. H. Lee: Protiva Biotherapeutics Inc.
Akin Akinc: Alnylam Pharmaceuticals Inc.
Birgit Bramlage: Alnylam Europe AG
David Bumcrot: Alnylam Pharmaceuticals Inc.
Matthew N. Fedoruk: Protiva Biotherapeutics Inc.
Jens Harborth: Alnylam Pharmaceuticals Inc.
James A. Heyes: Protiva Biotherapeutics Inc.
Lloyd B. Jeffs: Protiva Biotherapeutics Inc.
Matthias John: Alnylam Europe AG
Adam D. Judge: Protiva Biotherapeutics Inc.
Kieu Lam: Protiva Biotherapeutics Inc.
Kevin McClintock: Protiva Biotherapeutics Inc.
Lubomir V. Nechev: Alnylam Pharmaceuticals Inc.
Lorne R. Palmer: Protiva Biotherapeutics Inc.
Timothy Racie: Alnylam Pharmaceuticals Inc.
Ingo Röhl: Alnylam Europe AG
Stephan Seiffert: Alnylam Europe AG
Sumi Shanmugam: Alnylam Pharmaceuticals Inc.
Vandana Sood: Protiva Biotherapeutics Inc.
Jürgen Soutschek: Alnylam Europe AG
Ivanka Toudjarska: Alnylam Pharmaceuticals Inc.
Amanda J. Wheat: Protiva Biotherapeutics Inc.
Ed Yaworski: Protiva Biotherapeutics Inc.
William Zedalis: Alnylam Pharmaceuticals Inc.
Victor Koteliansky: Alnylam Pharmaceuticals Inc.
Muthiah Manoharan: Alnylam Pharmaceuticals Inc.
Hans-Peter Vornlocher: Alnylam Europe AG
Ian MacLachlan: Protiva Biotherapeutics Inc.

Nature, 2006, vol. 441, issue 7089, 111-114

Abstract: Making the most of RNAi Two papers this week highlight the impact of RNAi (RNA interference) in clinical medicine. Ngo et al. have developed a novel ‘Achilles heel' screen to identify genes that, if silenced, cause cancer cells to stop dividing. The novelty lies in a successful ‘negative’ screen that can reveal potential therapeutic targets that do not necessarily contain mutations or other alterations. Use of the screen on 2,500 genes in B-cell lymphoma cells identified three genes that were essential for cancer cell survival and growth of one particular B-cell lymphoma subtype. In particular the protein CARD11 looks a prime target. Zimmermann et al. report a significant step towards harnessing RNAi as a new class of drug treatment. They used systemic administration of small interfering RNA (siRNA) to silence a disease-causing gene in a non-human primate: it had previously been demonstrated in mice. Specifically, siRNA targeted against the gene for apolipoprotein B (ApoB) in cynomolgus monkeys successfully reduced in ApoB protein, serum cholesterol and low-density lipoprotein levels. This has implications for diseases associated with high cholesterol levels, such as coronary heart disease, and more broadly demonstrates that potential therapies may be developed against historically ‘non-druggable’ targets.

Date: 2006
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DOI: 10.1038/nature04688

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