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Biotech / Medical : Indications -- cardiovascular

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From: John McCarthy11/16/2004 8:32:40 AM
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Plunging levels

To test these souped-up siRNAs, the researchers chose, somewhat confusingly, to silence a gene called apoB that controls the level of cholesterol in blood.

When mice were injected with cholesterol-linked siRNAs designed to target apoB, the blood levels of the apoB protein dropped by nearly 70 per cent - and cholesterol levels also plunged. Non-cholesterol-linked siRNAs or siRNAs that were not perfect matches to the apoB sequence had no effect.

In the near term, siRNAs do not pose any serious competition for existing cholesterol-fighting drugs such as statins. It is not yet clear if the siRNAs’ effects last longer than 24 hours, for example, and the large doses needed would be extremely expensive.

But it appears the technique could be adapted to silence genes involved in a wide range of diseases. Although the apoB gene is only active in the liver and intestine, the modified siRNAs were also able to penetrate many other tissues, including heart, kidney, fat and lung.

Journal reference: Nature (vol 432, p 173)
Philip Cohen

newscientist.com
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