Viral and nonviral clinical trials have established proof-of-principle for gene transfer into the cystic fibrosis (CF) lung. It is currently unknown whether gene transfer efficiency is sufficient ...

A new mega-database of half a million mutations may flag new ways of treating genetic disease, scientists say.

In this review of RNA therapies, we paint an overview of what’s going on in a field that is addressing a number of diseases.

EYLEA HD® and EYLEA® remained the U.S. anti-VEGF category leader in 2024; aggregate U.S. net product sales were $6 billion for full-year 2024, up 1% based on preliminary (unaudited) results EYLEA HD ...

In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional. When the protein is not working correctly, it’s ...

Although newborn screening includes CF, this typically only covers a selection of common genetic variants, and with over 2000 reported in the cystic fibrosis transmembrane conductance regulator (CFTR) ...

Background Newborn bloodspot screening (NBS) for cystic fibrosis (CF) was introduced across the UK in ... from a dried bloodspot taken in the first week of life followed by DNA analysis. After 2007, ...

Various diseases and conditions related to heritable gene mutations can directly impact lifespan. These include cystic fibrosis and sickle cell anemia, to name a couple. Key Concepts in the Genetic ...