Hope is blooming in cystic fibrosis research.
Just one faulty gene can mean the difference between a healthy respiratory system or one marked by lung dehydration and mucous buildup blocking airways. Cystic fibrosis, a progressive genetic disorder, afflicts 30,000 people in the U.S., with a median life expectancy of just 40 years.
Now, gene therapy to fight the disease is getting a boost from a novel source: plants.
Gaurav Sahay, assistant professor of pharmaceutical sciences in the Oregon State College of Pharmacy, is exploring gene therapies using phytosterols — plant-based relatives of cholesterol — that change the shape of nanoparticles to help chemically modified genes get where they need to be inside cells. This nanoparticle approach causes cells to create the proteins needed to properly regulate chloride and water transport critical to healthy respiratory function.
Sahay’s findings could be used to make inhalable particles that can cross several barriers in the lungs of cystic fibrosis patients, enabling them to be treated with much greater efficacy. That has the potential to help thousands of people breathe easier.