In the 1980s, the discovery of the CFTR gene and mutations that cause CF further solidified the role of the sweat test in ...

The Gene Expression Core was founded in 1994 and promotes translational, multidisciplinary CF research by furnishing expertise concerning recombinant CFTR and other proteins relevant to CF disease ...

In their 2019 paper, Liu’s team used prime editing to alter the gene mutations causing sickle cell disease and Tay-Sachs disease. Liu explained that they attempted to edit mutated CFTR as well but ...

For this, they used a mouse line that carries a mutation in the CFTR gene. Intravenous injections of SORT LNPs carrying a gene editor targeted to this mutation resulted in its correction in almost ...

Take cystic fibrosis. It’s caused by a defect in the CFTR gene. But you need two copies of the defective gene to actually get the disease. So, if you only have one defective copy, you’re ...

By the time Enid Katz was two years old, she had already suffered through several bouts of bronchitis and was always hungry, ...

CF is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, resulting in abnormal chloride and sodium transport across epithelial cells. While advancements like Trikafta ...

in addition to maintenance of wild-type Sprague Dawley strain for additional use Generation and procurement of novel relevant CF animal models to understand newly developed CFTR modulators, and to ...

A Wicklow mother is among a concerned group of parents who are calling for individual trials of the modulator therapies for ...

“Alyftrek is our fifth CFTR modulator to secure FDA approval and represents another significant milestone in our journey to serially innovate and to improve the lives of people living with ...