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Cholesterol treatment to be tested

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A pioneering genetic technique has been used to lower cholesterol in monkeys, which scientists say could provide a new way of treating the condition in humans.

The study is the first demonstration of the approach, known as microRNA silencing, in non-human primates. Initial safety trials are now planned involving healthy human volunteers.

MicroRNAs (miRNAs) are short strands of RNA – a genetic molecule similar to DNA – that help regulate the way genes work. They are believed to play an important role in a variety of conditions from viral infections and heart disease to neurological disorders and cancer.

Scientists in the US and Denmark used injections of a particular molecule to block, or “silence”, liver miRNAs which look after cholesterol metabolism.

Their findings have been reported in the journal Nature.

Applying the treatment to African green monkeys led to a dose-dependent lowering of blood cholesterol. Three shots of the drug was sufficient to achieve efficient silencing of the target microRNA, known as miR-22.

The Danish pharmaceutical company leading the research now plans on trying out the same anti-miRNA compound on humans.

A Phase I clinical trial examining the safety of the drug is expected to get under way later this year.

Copyright © PA Business 2008

Nature






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