Gene-editing cystic fibrosis

Researchers have engineered a novel way to correct one of the genetic mutations that cause cystic fibrosis, a lethal disorder of the lungs and digestive system, as reported in the April issue of Nature Communications. Using gene-editing technology, the team combined healthy DNA with synthetic molecules called peptide nucleic acids that trigger repair near the cystic fibrosis mutation and then delivered the molecules in mice via biodegradable nanoparticles. They also tested the treatment on human cell cultures. “The percentage of cells in humans and in mice that we were able to edit the CFTR mutation in using these nanoparticles containing triplex forming peptide nucleic acids was higher than has been previously reported with any gene-editing technology,” said Marie E. Egan, M.D., professor of pediatrics and the study’s senior author. “This is step one in a long process. The technology could be used as a way to fix the basic genetic defect in cystic fibrosis.”