Gene Editing May Lead to Potential Treatments for Patients With Cystic Fibrosis

Gene editing may offer a promising treatment option for patients with cystic fibrosis (CF), according to a new research feature from the National Heart, Lung and Blood Institute (NHLBI).

CF, a genetic disease affecting more than160,000 people worldwide, is caused by mutations in the CFTR gene that lead to thick mucus buildup and reduced lung function. Although treatments like CFTR modulators have improved patient care, they are costly and ineffective in about 10% of CF patients, especially those with rare mutations.

To address these challenges, Paul McCray, MD, professor of pediatrics at the University of Iowa; and David Liu, PhD, of the Broad Institute, are leading efforts to enhance prime editing, a technique capable of making precise edits to the genetic code. In their recent NHBLI-supported study, they successfully used this technique to correct genetic mutations in CF lung and airway cells.

While the team’s work marks a step toward developing a one-time treatment that could benefit all individuals with CF, regardless of their genetic mutations, the investigators noted that challenges remain.

"The most challenging hurdle for gene editing techniques for lung diseases is to find efficient ways to deliver the technology to the right place in the body where it can work its magic," McCray said. "We have these amazing tools at our disposal, but how to get the tool to the target cells of interest is really where our focus is now."

McCray and his colleagues are actively testing different delivery systems in hopes of advancing these tools into clinical trials.