The Promise and Power of RNA

People whose bodies make an unusually active form of a certain protein tend to have dangerously high levels of cholesterol. Those with an inactive form of the protein have low cholesterol and a low risk of heart attacks.

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Needless to say, pharmaceutical companies would love to find a drug that can attach itself to the protein and block its activity. That might be difficult for this protein, which is called PCSK9. But a powerful new approach, called RNA interference, may surmount that obstacle. Instead of mopping up a protein after it has been produced, as a conventional drug would do, RNA interference turns off the faucet, halting production of a protein by silencing the gene that contains its recipe.

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In monkeys, a single injection of a drug to induce RNA interference against PCSK9 lowered levels of bad cholesterol by about 60%, an effect that lasted up to three weeks. Alnylam Pharmaceuticals, the biotechnology company that developed the drug, hopes to begin testing it in people next year.

The drug is a practical application of scientific discoveries that are showing that RNA, once considered a mere messenger boy for DNA, actually helps to run the show. The classic, protein-making genes are still there on the double helix, but RNA seems to play a powerful role in how genes function.

“This is potentially the biggest change in our understanding of biology since the discovery of the double helix,” said John Mattick, a professor of molecular biology at the University of Queensland in Australia. And the practical impact may be enormous.

RNA interference, or RNAi, discovered only about 10 years ago, is attracting huge interest for its seeming ability to knock out disease-causing genes. There are already at least six RNAi drugs being tested in people, for illnesses including cancer and an eye disease. And while there are still huge challenges to surmount, that number could easily double in the coming year.

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Sources: The Times Of India

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