Health Headlines: Scientists testing new Cystic Fibrosis treatment

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Published: Feb. 3, 2023 at 11:57 AM CST
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NEW HAVEN, Conn. (Ivanhoe Newswire) - Cystic Fibrosis is an inherited disease that affects the lungs, pancreas, and other organs. There is no cure but new medications have come a long way toward improving the symptoms for many patients. But for some, the current medications are insufficient so scientists are working to change that.

Deep breaths, in and out. It’s something that many of us take for granted. But for those with cystic fibrosis, their lungs fill with thick mucus, making it tough to get air. That mucus also ends up clogging the pancreas and intestines multiplying the victim’s problems.

Cystic Fibrosis expert Dr. Marie Egan is studying ways to help the body repair the mutated DNA that causes the condition.

She and her Yale colleagues have 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.

“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.

“I think it’s imperative that we move forward, and not just for CF, but for all genetic disorders.”

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.

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