Using these RNA-delivery particles, researchers hope to develop new treatments for cystic fibrosis and other lung diseases. MIT and University of Massachusetts Medical School engineers have developed nanoparticles that deliver messenger RNA encoding useful proteins to the lungs, with potential ap
Engineers at MIT and the University of Massachusetts Medical School have designed a new type of nanoparticle that can be administered to the lungs, where it can deliver messenger RNA encoding useful proteins. Credit: iStock, edited by MIT News-delivery particles, researchers hope to develop new treatments for cystic fibrosis and other lung diseases.
“This is the first demonstration of highly efficient delivery of RNA to the lungs in mice. We are hopeful that it can be used to treat or repair a range of genetic diseases, including cystic fibrosis,” says Daniel Anderson, a professor in MIT’s Department of Chemical Engineering and a member of MIT’s Koch Institute for Integrative Cancer Research and Institute for Medical Engineering and Science .
Several years ago, Anderson’s lab set out to design particles that would be better able to transfect the epithelial cells that make up most of the lining of the lungs. In 2019, his labencoding a bioluminescent protein to lung cells. Those particles were made from polymers instead of lipids, which made them easier to aerosolize for inhalation into the lungs. However, more work is needed on those particles to increase their potency and maximize their usefulness.
After one dose of mRNA, about 40 percent of lung epithelial cells were transfected, the researchers found. Two doses brought the level to more than 50 percent, and three doses up to 60 percent. The most important targets for treating lung disease are two types of epithelial cells called club cells and ciliated cells, and each of these was transfected at about 15 percent.
“This achievement paves the way for promising therapeutic lung gene delivery applications for various lung diseases,” says Dan Peer, director of the Laboratory of Precision NanoMedicine at Tel Aviv University, who was not involved in the study. “This platform holds several advantages compared to conventional vaccines and therapies, including that it’s cell-free, enables rapid manufacturing, and has high versatility and a favorable safety profile.
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