Study provides proof in humans of RNA interference using targeted nanoparticles

Posted: April 4, 2010 at 10:56 pm

Story Summary: Moreover, the team provided the first demonstration that this new type of therapy can make its way to human tumors in a dose-dependent fashion, meaning a higher number of nanoparticles sent into the body results in a higher number of nanoparticles found in the tumor cells. Published in the March 21 advance online edition of the journal Nature, the results demonstrate the feasibility of using both nanoparticles and RNA interference-based therapeutics in patients. RNAi is a new way to stop the production of proteins, researchers said. Even before the discovery of RNAi, Davis and his team had begun working on ways to deliver nucleic acids into cells through systemic administration. They eventually created a four-component system featuring a unique polymer that can self-assemble into a targeted, siRNA-containing nanoparticle. These nanoparticles are able to take the siRNAs to the targeted site within the body, Davis said. In addition, Davis and his colleagues were able to show that the higher the nanoparticle dose administered to the patient, the higher the number of nanoparticles found in tumor cells — the first example of dose-dependent response using targeted nanoparticles. Even better, Davis said, the results showed the siRNAs had done their job. In the tumor cells analyzed by the researchers, the mRNA encoding the cell-growth protein ribonucleotide reductase had been degraded. Its the first time anyone has found an RNA fragment from a patients cells showing the mRNA was cut at exactly the right base via the RNAi mechanism, Davis said. There are many cancer targets that can be efficiently blocked in the laboratory using siRNA, but blocking them in the clinic has been elusive, said the studys senior author, Dr. Antoni Ribas, an associate professor of medicine and surgery and a researcher at UCLAs Jonsson Comprehensive Cancer Center. This is because many of these targets arent amenable to be blocked by traditionally designed anti-cancer drugs, said Ribas, who ran the Phase 1 clinical trial….Read the Full Story

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