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10 Emerging Technologies That Will Change Your World

(Page 8 of 11)

RNAi Therapy

From heart disease to hepatitis, cancer to AIDS, a host of modern ailments are triggered by our own errant genes-or by those of invading organisms. So if a simple technique could be found for turning off specific genes at will, these diseases could-in theory-be arrested or cured. Biochemist Thomas Tuschl may have found just such an off switch in humans: RNA interference (RNAi). While working at Germany’s Max Planck Institute for Biophysical Chemistry, Tuschl discovered that tiny double-stranded molecules of RNA designed to target a certain gene can, when introduced into human cells, specifically block that gene’s effects.

Tuschl, now at Rockefeller University in New York City, first presented his findings at a meeting in Tokyo in May 2001. His audience was filled with doubters who remembered other much hyped RNA techniques that ultimately didn’t work very well. “They were very skeptical and very critical,” recalls Tuschl. What the skeptics didn’t realize was that RNAi is much more potent and reliable than earlier methods. “It worked the first time we did the experiment,” Tuschl recalls. Within a year, the doubts had vanished, and now the technique has universal acceptance-spawning research at every major drug company and university and likely putting Tuschl on the short list for a Nobel Prize.

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