Nanotechnology encapsulation delivers RNA interference agents 10-fold more effectively

RNA interference (RNAi) is a way to decrease expression of a specific gene without otherwise affecting the cell, and it therefore could be a very promising treatment for a wide variety of diseases—if it could be reliably delivered into the diseased cell cytoplasm. One possible nanotech solution to this problem takes the form of a 10-fold more effective delivery of RNAi protected in nanocapsules formed by novel lipid-like molecules. From “Nano RNA delivery: Novel delivery agents could mean a more targeted way to turn off disease genes” at Technology Review, written by Kevin Bullis (credit to

An experimental and potentially powerful way to fight disease, called RNA interference (RNAi), could now be closer to reality, as researchers at MIT and Alnylam, a biotech company based in Cambridge, MA, have addressed a key obstacle to effectively delivering the treatment to targeted cells. The researchers report a method for quickly synthesizing more than a thousand different lipid-like molecules and screening them for their ability to deliver short RNA molecules to cells. They’ve shown that some of these delivery agents are 10 times as effective at delivering RNA than previous methods were.

…In tests on mice, the best of these delivery agents were 10 times as effective at delivering RNA to treat a respiratory ailment, compared with existing methods that deliver the RNA directly to the lungs without it being encapsulated. The researchers also demonstrated that the agents worked for delivering RNA through the bloodstream to the liver and various tissues. What’s more, initial tests in primates showed promising results.

…The researchers are working with Alnylam, which will be moving the delivery agents toward clinical trials. They’re also continuing to sort through new variants of the molecules to find ones that work better, and they’re testing the effectiveness of the delivery agents for targeting different diseases and for delivering different therapeutics in addition to RNA.

The research was published in Nature Biotechnology (abstract)

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