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Sanford-Burnham researchers develop novel nanoparticle to deliver powerful RNA interference drugs
Date:7/9/2013

siRNA molecules. In their experiment, Dr. Rana and colleagues showed in the lab that iNOP-7DS is reducible that is, the disulfide bonds holding the siRNA molecules can be broken.

They next showed that iNOP-7DS can be delivered effectively inside cultured murine liver cells, where the siRNA molecules silenced a gene called ApoB. This gene has been notoriously difficult to regulate in liver cells with small molecule drugs; high levels of the protein that ApoB encodes can lead to plaques that cause vascular disease.

Dr. Rana's lab further showed in tests that their nanoparticle remained stable in serum, suggesting that it is not degraded in the bloodstream. Finally, the researchers showed in tests with mice that their nanoparticle iNOP-7DS can be delivered effectively to the liver, spleen, and lung; and it suppressed the level of messenger RNA involved in the expression of the ApoB gene. In their in vivo experiment, they found that extremely small doses of siRNA were effective.

The next step, Dr. Rana said, is to increase the efficacy of iNOP-7DS in other in vivo experiments. "We would like to target not only ApoB, but cancer causing genes as well and in other tissues. That is the next goal." By marshaling the naturally occurring phenomenon of RNA interference, scientists are developing new ways to silence errant gene expression involved in illnesses. The nanoparticles developed by Dr. Rana and colleagues offer a potential new strategy for delivering this powerful therapeutic approach.


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Contact: Deborah Robison
drobison@sanfordburnham.org
407-615-0072
Sanford-Burnham Medical Research Institute
Source:Eurekalert

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