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

for the treatment of cystic fibrosis in patients with at least one F508del mutation or another treatment-responsive mutation in the CFTR gene. The fixed-dose triple-combination therapy ...

The CFTR gene was identified in 1989, but few companies jumped at the chance to use the information to develop new therapies, because the market was perceived as too small. It is therefore ...

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

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 ...

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 ...

The CFTR Protein Distribution Core offers high-quality, full-length recombinant cystic fibrosis transmembrane conductance regulator (CFTR) proteins expressed in and purified from mammalian cells. Wild ...

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

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 ...

View Full Profile. Learn about our Editorial Policies. 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 ...

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 ...

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 unaffected.