An optimized version of prime editing technology raises the possibility of a one-time treatment for cystic fibrosis. In their 2019 paper, Liu’s team used prime editing to alter the gene mutations ...

Cystic fibrosis is among the most common, known and studied genetic diseases. It affects over 100,000 people worldwide and reduces life expectancy mainly as it causes lung and respiratory problems.

Affecting an estimated 100,000 people globally, cystic fibrosis (CF) cases stem from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In the past several decades, ...

Researchers at the University of Trento have developed a CRISPR-based adenine base editing therapy that repaired the previously untreatable CFTR 1717-1G>A mutation in patient-derived cell models.

Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) is a combination medicine approved by the FDA in 2024. Alyftrek is approved to treat people ages 6 and older who have ...

The cystic fibrosis transmembrane conductance regulator (CFTR) is a complex protein that helps maintain fluid balance in several organs. Mutations in the CFTR protein can lead to symptoms of cystic ...

The European Medicines Agency (EMA) recommended in its April meetings approval of marketing authorizations for two orphan medicines: Alyftrek for the treatment of cystic fibrosis and Attrogy for the ...

A faulty gene has been found to disrupt the enzyme balance in lung cells, resulting in mucus deposits and inflammation Coughing and spluttering is a familiar soundtrack to winter, but for people with ...

Please provide your email address to receive an email when new articles are posted on . Sweat chloride did not significantly differ between baseline and day 28 of triple therapy in this patient ...

Breathing disorders are common in the U.S. with conditions like COPD affecting some 7% of the population and asthma affecting another 10%. Digestive conditions like GERD, acid reflux, Celiac disease ...