Potentially one of the biggest breakthroughs in medicine.
Scientists have found a way of treating potentially fatal diseases by switching off harmful genes. In what is described as one of the most important breakthroughs in decades, researchers have shown that RNA interference can cut cholesterol levels in laboratory mice with a method that could be applied to humans at risk of heart attacks.
They say RNA interference (RNAi) could be used to treat a wide range of disorders, from HIV and Aids to genetic diseases and cancer. RNAi can switch off harmful genes that cause disease but leave other essential genes untouched.
Science had not previously demonstrated a safe, reliable way of using it on patients, but now researchers led by Hans-Peter Vornlocher, head of research at the pharmaceuticals company Alnylam Europe, have devised a simple method of delivering RNAi to all the cells of the body via an intravenous injection. In experiments on mice they injected short lengths of RNA - a molecule similar to DNA - that had been designed to switch off or "silence" the gene responsible for producing apoliprotein B, a protein involved in the synthesis of the damaging form of cholesterol.
By coincidence, the researchers used another form of the cholesterol molecule, which they had attached to the RNA molecule to allow the RNA to slip through the cell membranes of the body.