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 ...

Other gene therapy possibilities could include manipulating the CFTR gene in people with cystic fibrosis, or for treating rare genetic diseases. LNPs can also carry small interfering RNA (siRNA ...

Cystic fibrosis is caused by faults in a gene called CFTR, which gives the body “instructions” to produce a protein that ...

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 ...

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 ...

Some examples of genetic diseases linked to cytosine mutations include: Sickle cell anemia: A single C-to-T mutation in the beta-globin gene leads to the production of abnormal hemoglobin, causing red ...

Other rare mutations that are not eligible for CFTR modulators will be explored as the editing technology matures. “Gene editing is a promising, complex technology, which will take many years to ...

CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. An estimated 105,000 people have been diagnosed with CF across 94 countries, according to the CF ...