Yale collaborators use gene editing tech to fix a cystic fibrosis mutation

Cystic fibrosis cells show better uptake (left to right) after gene correction.--Courtesy of Yale

A project at Yale successfully adapted gene editing technology to correct the genetic mutation that is most commonly linked to cystic fibrosis.

Using human and mouse cells, the team used synthetic peptide nucleic acids--or PNAs, which are similar to DNA--along with donor DNA to "edit" the F508del mutation that is the most common cause of CF.

"What the PNA does is clamp to the DNA close to the mutation, triggering DNA repair and recombination pathways in cells," noted Marie Egan, professor of pediatrics and of cellular and molecular physiology. Egan was part of a collaborative team that included Peter Glazer, chair of therapeutic radiology, and Mark Saltzman, chair of biomedical engineering.

Significantly, the team used microscopic nanoparticles specifically designed to penetrate target human and mouse cells in their research.

"The percentage of cells in humans and in mice that we were able to edit was higher than has been previously reported in gene editing technology," said Egan.

The study was published April 27 in Nature Communications.

- here's the release
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