RNA-enabled nanoparticles could treat liver disease

Massachusetts Institute of Technology engineers have fashioned a smarter nanoparticle that runs RNA interference as a means of disease modification, the institute announced Feb. 10.

The particles transport genetic material that switches off genes involved in the disease process, particularly in the case of cancer and liver diseases. These particles avoid previous side effects because the short RNA strands are swaddled in a fatty sphere. The new lipoprotein-coated particles have so far led to more silencing of targeted genetic expression than all former attempts.

One particular compound, called cKK-E12, led to five times more quieting of targeted genes than any other delivery vehicle from the literature.

“That’s important because we don’t want the material to silence all the targets in the human body,” said Yizhou Dong, a researcher at the Koch Institute via a press release. “If we want to treat patients with liver disease, we only want to silence targets in the liver, not other cell types.”

The research is at the preclinical stage, but further pharmacokinetic studies could one day usher RNA nanomedicine into human studies. Right now the applications are limited to liver disease, as that is where these particles naturally accumulate. The method has seen complications when applied to other clinical applications, but some success has been seen in the area of amyloid-influenced disease processes. 

“We really wanted to develop materials for clinical use in the future,” remarked Dong. “That’s our ultimate goal for the material to achieve.”