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Gene-Targeted Cancer Fix Could Be a Breakthrough
Date:3/21/2010

For first time in humans, scientists used RNA to stop production of protein driving malignancy

SUNDAY, March 21 (HealthDay News) -- For the first time in humans, scientists have successfully used a gene-manipulation therapy to enter tumor cells and block the production of toxic proteins that are causing cancer, researchers report.

"They're basically putting an instruction booklet into the cell saying, 'We don't want this protein expressed for now,'" explained Gregory Adams, co-leader of the developmental therapeutics program at Fox Chase Cancer Center in Philadelphia. "It's pretty amazing. It's potentially huge."

"This is something we've been waiting to see," he continued.

"This directly interferes with the genetic mechanisms that promote cancer to stop the production of a particular protein," added Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society. "This is one step away from getting into the actual DNA."

As reported online March 21 in Nature, this is the first time the process, known as RNA interference (RNAi), has been shown to work in humans.

The process of RNA interference involves putting two strands of RNA together to form so-called "small interfering RNAs" (siRNAs) and inserting them into cells. Once there, these interlopers cut the messenger RNA (mRNA) that is ordinarily used to make specific proteins. This discovery won the Nobel Prize in 2006.

But the work that nabbed the prize was done in worms -- a far cry from humans.

And there were other challenges, not the least of which was how to get the siRNAs into the appropriate cell and then make sure they did what they were supposed to do.

This team, from the California Institute of Technology (CalTech), devised a super-small nanoparticle system that, when injected into the body, would make its way to the tumor, deposit the siRNAs into the tumor cell and leave them to
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