Best Life: Gene therapy for cystic fibrosis

Published: Feb. 17, 2023 at 5:53 AM CST
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NEW HAVEN, Conn. (Ivanhoe Newswire) – Cystic fibrosis is an inherited disease that affects the lungs, the pancreas, and other organs. There is no cure. New medications have come a long way to improve the symptoms for many patients, but not everyone. Now, researchers at Yale University are studying a therapy that could correct the condition before symptoms start.

Deep breaths in and out, something many of us take for granted. But for people with cystic fibrosis, the lungs fill with thick mucus, making it tough to get air. Mucus also clogs the pancreas and intestines.

Pediatric pulmonologist at Yale Medicine, Marie Egan, MD is studying ways to help the body repair the mutated DNA that causes the condition.

Dr. Egan and her Yale colleagues developed a new gene editing technique that targets a mutation in the CFTR gene. They’ve designed small molecules that can be delivered into the body intravenously.

Dr. Egan explains, “We include a small piece of DNA or blueprint that has the right instructions. Your own body can fix itself.”

Dr. Egan says the molecules would be administered as a one-time therapy and could be a delivery method and treatment for other conditions.

Scientists tested the technique in the lab and found that it worked not only in the lungs, but other organs that are affected by cystic fibrosis. The researchers say the technique needs to be tested in humans, but they suspect it could be successfully administered at any stage of life, not just in babies, but right after the condition is diagnosed. Cystic fibrosis is one of the conditions most states screen for during newborn testing.

Contributors to this news report include: Cyndy McGrath, Producer; Kirk Manson, Videographer; Roque Correa, Editor.

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