Editor's Summary
17 April 2008
'Therapeutic' microRNA
The realization that microRNAs play a central role in gene regulation in both normal development and disease, such as cancer, cardiac disease and metabolic disorders, suggests that they might be viable targets for therapeutic intervention. Now, for the first time, efficient, long-lasting and reversible microRNA silencing has been achieved in non-human primates. African green monkeys received intraperitoneal injections of a short modified DNA sequence that binds to and blocks the function of miR-122, a microRNA that regulates genes that influence cholesterol levels. Dose-dependent lowering of blood cholesterol followed, with no signs of toxicity. It is still a giant leap to therapeutic applications and, as microRNAs can act on many genes, toxicity risks are considerable.
Authors: Making the paper: Sakari Kauppinen
Therapies that target small RNA molecules show promise in animals.
doi:10.1038/7189xa
Letter: LNA-mediated microRNA silencing in non-human primates
Joacim Elmén, Morten Lindow, Sylvia Schütz, Matthew Lawrence, Andreas Petri, Susanna Obad, Marie Lindholm, Maj Hedtjärn, Henrik Frydenlund Hansen, Urs Berger, Steven Gullans, Phil Kearney, Peter Sarnow, Ellen Marie Straarup & Sakari Kauppinen
doi:10.1038/nature06783
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