Researchers take key step toward improving treatment of cystic fibrosis

Researchers at Oregon State University and Oregon Health & Science University have taken a key step toward improving and lengthening the lives of cystic fibrosis patients, who experience chronically clogged airways and a dramatically shortened life expectancy.

The team of scientists and clinicians has engineered inhalable lipid nanoparticles that can effectively deliver messenger RNA to the lungs, prompting lung cells to manufacture the protein that thwarts the disease.

Findings were published in ACS Nano.

The research was led by postdoctoral scholar Jeonghwan Kim and Gaurav Sahay, an associate professor of pharmaceutical sciences in the OSU College of Pharmacy who studies lipid nanoparticles, or LNPs, as a gene delivery vehicle with a focus on cystic fibrosis. Lipids are fatty acids and similar organic compounds including many natural oils and waxes, and nanoparticles are tiny pieces of material ranging in size from one- to 100-billionths of a meter.

Cystic fibrosis is a progressive genetic disorder that results in persistent lung infection and affects 30,000 people in the U.S., with about 1,000 new cases identified every year. More than three-quarters of patients are diagnosed by age 2, and despite steady advances in alleviating complications, the median life expectancy is still just 40 years.

One faulty gene — the cystic fibrosis transmembrane conductance regulator, or CFTR — causes the disease, which is characterized by lung dehydration and mucous buildup that blocks the airway.

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