I’ve written about RNA interference often and will in the future. After spending weeks studying and talking to leaders in the field, I have to admit that I’m boggled at the rate of progress. It’s a sign of these accelerating times that the breakthrough research in RNAi was published only 10 years ago by Craig Mello and Andrew Fire. In 2006, they won their Nobel Prize in physiology or medicine. This is, as far as I can tell, the shortest time from discovery to Nobel in history.

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

Patrick Cox

Patrick Cox has lived deep inside the world of transformative technologies for over 25 years. In the 1980s, he worked in computer software development and manufacturing. By the mid-1990s, he worked as a consultant for Netscape — the company that handled 90% of all Internet browsing traffic at the time. InfoWorld and USA Today have featured Patrick’s research many times. He’s also appeared on Crossfire and Nightline. This expertise bought him to Agora Financial, where he now heads Breakthrough Technology Alert, the only place you’ll find the truly transformational technologies that offer exponential gains.

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