Children whose cystic fibrosis is caused by a rare gene variant are excluded from a new treatment which could potentially ...

The Gene Expression Core was founded in 1994 and promotes translational, multidisciplinary CF research by furnishing expertise concerning recombinant CFTR and other proteins relevant to CF disease ...

Cystic fibrosis is a chronic genetic disease affecting both the lungs and the gastrointestinal system. It is caused by a gene defect that is passed on to children having parents who carry a ...

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 ...

honed on the rare single-gene disorders, such as Huntington disease (HD) and cystic fibrosis (CF), which dominated the field in the last century (see Genetic Testing Timeline). "Geneticists today are ...

Cystic fibrosis (CF) is a single-gene disorder with insufficient treatment options and a target organ, the lung that is relatively easily accessible. Thus, it is not surprising that in the early ...