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RNAi research to turn off disease-causing genes offers new hope

Phillip D. Zamore is codirector of the RNA Therapeutics Institute.
Phillip D. Zamore is codirector of the RNA Therapeutics Institute.UMass Medical School

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In 1998, two researchers made a startling discovery with enormous potential: They could attack diseases by turning off the genes that provide them with their malevolent energy.

The scientists, from the University of Massachusetts Medical School and Stanford University, had come up with a way to block, or “silence,” genes by using strands of ribonucleic acid, or RNA, to interfere with the genes’ normal production of proteins. Remove the proteins and the diseases fed by them would go away.

But by the time Craig Mello and Andrew Z. Fire were rewarded for their breakthrough with the Nobel Prize in Medicine in 2006, their big idea, RNA interference, had failed to produce breakthroughs.

But “there’s been significant progress” over the past decade, said Andre Turenne, at Genzyme, which recently signed a development deal with Alnylam Pharmaceuticals.

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