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Translating promising science into new therapies.
Amid the national debate over stem cells and therapeutic cloning, there's another biomedical technology that is showing increasing promise without the ethical conundrums. RNA interference (RNAi) is a natural regulatory process in which small, double-stranded RNA molecules turn off specific genes in a cell. Because RNAi is highly targeted and efficient, it has become a widely used tool for understanding what genes do and how they work. But its real payoff lies in new therapies -- and developing them will require a renewed commitment to funding and research.
Exploiting the properties of RNA molecules could yield more-effective drugs to fight cancer, HIV, influenza, and other diseases. Rather than blocking the effects of specific proteins, which is what conventional drugs do, an RNAi-based therapy could in theory stop the proteins from being made in the first place. Mark Kay, director of the Program in Human Gene Therapy at the Stanford University School of Medicine, calls RNAi "incredibly robust technology with incredible therapeutic potential" but cautions that it's in a "very new, very early stage."
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