CFTR is a protein essential to wet surfaces throughout your body, like the airways of your lungs. People with cystic fibrosis have a gene mutation that causes problems with the protein. Some ...

The scientific imperative to identify drugs that could repair mutant CFTR presented a formidable challenge. Small molecules that specifically bound and repaired a defective CFTR peptide fold were ...

Scientists at St. Jude Children's Research Hospital and Rockefeller University have combined their expertise to gain a better understanding of the cystic fibrosis transmembrane conductance regulator ...

Care for patients with cystic fibrosis has undergone transformative changes over the past decade and serves as an example of how an understanding of the functional consequences of a genetic disease ...

Genetic testing plays an important role in cystic fibrosis screening and diagnosis. It can help determine if a person has cystic fibrosis or carries gene mutations that cause it. Other tests can also ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...