Nanoparticles Offer New Directions in Treatment of Cystic Fibrosis

Researchers successfully use liquid nanoparticles to deliver gene-editing tools to all lung cell types in mice

We've come a long way in the treatment of cystic fibrosis, a genetic disorder that affects the cells, glands and tissues that produce mucous and sweat. However, there are still some patients who aren't eligible for these treatments.

Fortunately, researchers are attempting to open up new avenues using miniaturized molecules made of fat and the gene editing technology CRISPR. In a recently published study in Science, scientists from the University of Texas Southwestern Medical Center, showed that the liquid nanoparticles (LNPs) could deliver intravenously gene editing tools to lung cells in mice. They found that this approach enabled long-lasting gene correction in a mouse model of CF and in patient cells.

The scientists used a strategy called selective organ sorting, to ensure that the engineered LNPs carrying CRISPR-based edited machinery reached their target. Indeed, an analysis of lung tissue from mice treated with the IV gene therapy drug showed that the delivery system remained durable 22 weeks after administration.

To read more about this research development check out the entire story in The Scientist.