New nanoparticles can perform gene editing in the lungs
Using these RNA-delivery particles, researchers hope to develop new treatments for cystic fibrosis and other lung diseases.
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.
In a study of mice, Daniel Anderson, a professor in MIT’s Department of Chemical Engineering and his colleagues used the particles to deliver mRNA encoding the machinery needed for CRISPR/Cas9 gene editing.
The 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 negatively charged mRNA, and it also help mRNA to escape from the cellular structures that engulf the particles once they enter cells.
The lipid tail structure, meanwhile, helps the particles to pass through the cell membrane. The researchers came up with 10 different chemical structures for the lipid tails, along with 72 different head groups. By screening different combinations of these structures in mice, the researchers were able to identify those that were most likely to reach the lungs.
To deliver the particles in this study, the researchers used a method called intratracheal instillation, which is often used as a way to model delivery of medication to the lungs. They are now working on making their nanoparticles more stable, so they could be aerosolized and inhaled using a nebulizer.
The researchers also plan to test the particles to deliver mRNA that could correct the genetic mutation found in the gene that causes cystic fibrosis, in a mouse model of the disease. They also hope to develop treatments for other lung diseases, such as idiopathic pulmonary fibrosis, as well as mRNA vaccines that could be delivered directly to the lungs.
https://news.mit.edu/2023/new-nanoparticles-can-perform-gene-editing-lungs-0330
ارسال به دوستان