RNAi is the cellular mechanism that blocks invaders such as viruses that hijack our genetic mechanisms to reproduce. By harnessing this defense, we can “silence,” or interfere, with the actions of any gene in the body.

This is important because most diseases are caused by these gene actions, called gene expression. Specifically, they control proteins that cause and cure specific diseases. This is the power to cure a disease as if by flipping a switch. RNAi scientists are racing to develop ways to control the production of these proteins.

There are other benefits associated with this direct control of gene expression.

Doses may be extremely small, and the substances themselves are nontoxic. Both of these factors reduce costs and traditional concerns about side effects caused by conventional small molecule medicines.

The list of diseases susceptible to RNAi cures includes cancers, HIV, arthritis, arteriosclerosis and most hereditary disorders. This is a breakthrough with few equals in the history of science.

There are still details to be worked out, of course. The genes that control specific diseases have to be identified. Then, the right RNA sequence has to be delivered to the disease-creating gene. This is not, however, as daunting as it may sound. Tests on numerous genes have proven the concept.

Until now, the delivery issue has proven the biggest hurdle. RNA interfering sequences work, but they are fragile and can be destroyed by our own biological defenses before they have a chance to work their cures. Nevertheless, new means of delivery are continually coming to light.

Regards,
Patrick Cox

October 24, 2008

Revolutionary Cure for Everything from Cancer to HIV was originally featured in the Penny Sleuth.