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“Tiny RNA genes may be the biological equivalent of dark matter-all around us but almost escaping detection,” wrote Gary Ruvkun, a Harvard Medical School molecular biologist, in 2001 in the journal Science. What are these mysterious genes doing? “I suspect what we’re looking at is a very ancient method of controlling gene expression,” says Zamore.

If microRNAs are switches that decide whether stem cells become neurons or muscle, or whether cancer cells grow or die, then RNA interference is a lot more important than anyone imagined just a few years ago. “We simply stumbled upon a whole new branch of molecular biology that we didn’t know about before,” says Michigan’s Engelke.

To the optimists, these breakthroughs portend the quick development of effective drugs. And even biomedical researchers made cynical by extravagant claims for magical cures think RNAi just may be the real thing. Last year, when RNAi first worked in human cells, “everyone woke up and said, I wonder if this is the silver bullet?’” says Engelke. “And it might be. It might be.”

Companies Developing RNA InterferenceCOMPANYPRIMARY RNAi FOCUSAlnylam Pharmaceuticals
(Cambridge, MA)
Therapeutics Benitec
(Brisbane, Australia)
Intellectual property for genomics and therapeutics Cenix BioScience
(Dresden, Germany)
Drug target identification and therapeutics for cancer Devgen
(Ghent, Belgium)
Drug target identification and therapeutics for diabetes, depression, and Parkinson’s disease Genetica
(Cambridge, MA)
Drug target identification for cancer Mirus
(Madison, WI)
Therapeutics, using long double-stranded RNA Ribopharma
(Kulmbach, Germany)
Therapeutics for cancer and hepatitis C

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