BI 3720931 is designed to insert the CFTR gene’s functional copy directly into the airway epithelial cells’ DNA. Credit: Andrey_Popov / Shutterstock. Boehringer Ingelheim, the UK Respiratory Gene ...

A new inhalable medicine, potentially improving lung disease in all cystic fibrosis patients, is in human trials across the UK and Europe (1 Trusted Source A Study to Test How Well BI ...

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 ability ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Until recently, drug development efforts have emphasised therapies treating downstream signs and symptoms resulting from the underlying CF biological defect: reduced function of the CF transmembrane ...

There are many misconceptions about CF. Learn the facts on our page, Dispelling Misconceptions About Cystic Fibrosis. In people with CF, mutations in the cystic fibrosis transmembrane conductance ...

has become a standard of care therapy for CF patients who have at least one F508del mutation in the CFTR gene. This drug, approved in 2019, has also become the company’s top-selling product.

The management of CF has been changing drastically over the last few years, especially after the introduction of CFTR modulators. Currently, 188,336 people have the disease in 96 countries, but only ...

We applied a strategy of serial screening steps to 45 patients with congenital absence of the vas deferens and characterized cystic fibrosis transmembrane conductance regulator gene mutations in ...

2 With the discovery of the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) and readily available laboratory techniques to characterise the common mutations, it was anticipated ...

The receiver operating characteristic (ROC) curve affirmed the model’s robust predictive capability. Validation of the model was performed in the GSE190826 cohort and our institution’s cohort. Gene ...