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"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.

With further development, these particles could offer an inhalable treatment for cystic fibrosis and other diseases of the lung, the researchers say.

“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 NAME_1, 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 (IMES).

In a study of mice, NAME_2 and his colleagues used the particles to deliver mRNA encoding the machinery needed for CRISPR/Cas9 gene editing. That could open the door to designing therapeutic nanoparticles that can snip out and replace disease-causing genes.

The senior authors of the study, which appears today in Nature Biotechnology, are NAME_2; NAME_3, the David NAME_4. Koch Institute Professor at MIT; and NAME_5, an associate professor at the UMass Medical School RNA Therapeutics Institute. NAME_6, a former MIT postdoc who is now an assistant professor at the University of Toronto; NAME_7, an MIT postdoc; and NAME_8, a postdoc at UMass Medical School, are paper’s lead authors.

NAME_9 Avoid using the following keywords: gene, mrna, rna, integrative
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