ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA ...

Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

the protein it produces will be flawed and cause mucus to thicken abnormally throughout the body. In recent years, scientists have developed drugs called CFTR modulators that can improve CFTR ...

A MIRACLE pill that’s been shown to help with many of the symptoms of cystic fibrosis (CF) has been approved for thousands of ...

One reason for that is that around 2,000 mutations in the CFTR gene can lead to CF, a disease caused by an imbalance in chloride and sodium ions in the body that results in thick, sticky mucus ...

Cystic fibrosis (CF) is caused by defects in the CFTR gene, which causes a buildup of thick sticky mucus in the lungs and digestive system. It causes lung infections and gradually affects the ...

In March 2025, Vertex Pharmaceuticals Incorporated announced a study is to evaluate the safety, and tolerability and efficacy ...

Researchers introduced functional porosomes into the plasma membrane of CFTR mutant cells to demonstrate that porosome reconstitution normalizes mucus secretion more than twice as effective than ...

Cystic fibrosis is a genetic disorder driven by CFTR mutations that affects the exocrine glands. Manifesting with excessively viscous mucus production, patients typically experience obstruction of ...

Cystic fibrosis (CF) is caused by defects in the CFTR gene, which causes a buildup of thick sticky mucus in the lungs and digestive system. It causes lung infections and gradually affects the ...

The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally ...