Scientists create nanoparticles for gene therapies in the lungs

Messenger RNA has great therapeutic potential to treat various diseases caused by defective genes. Until now, one of the obstacles to its application has been how difficult it is to deliver it to the correct part of the body, without unwanted side effects.

Daniel Anderson’s lab, at the Massachusetts Institute of Technology (MIT) in the United States, set out to develop lipid nanoparticles that could be delivered to the lungs. Particles are made up of molecules that contain two parts: a positively charged head group and a long lipid tail.

The positive charge of the head group helps the particles to interact with the negatively charged mRNA, and also helps the mRNA to escape from cell structures that engulf the particles once they enter cells.

The lipid structure of the tail, for its part, helps the particles to cross the cell membrane.

With further development, these particles could offer an inhalation-applicable treatment for cystic fibrosis and other lung diseases.

This is the first demonstration of highly efficient delivery of RNA to the lungs, so far only in mice. “We are hopeful that it could be used in humans to treat or repair various genetic diseases, including cystic fibrosis,” Anderson says.

Artist’s recreation of nanoparticles capable of modifying genes. (Image: Amazings/NCYT)

Another advantage of the new nanoparticles is that they break down quickly, allowing them to be removed from the lung in a few days and reducing the risk of inflammation.

The new nanoparticles could also be administered multiple times to the same patient if a repeat dose is necessary. This makes them more advantageous than another messenger RNA delivery method, which uses a modified version of harmless adenoviruses. These viruses are very efficient at delivering RNA, but cannot be delivered repeatedly because they induce an immune response in the host. (Fountain: NCYT by Amazings)