Modulator drugs interact with mutant CFTR and force abnormally-folded proteins into a more functional structure. But these modulators are mutation-specific, and Skach estimates that 7% of cystic ...
Heike Pahl (Univ. Hospital, Freiburg, Germany) reported that expression of mutant CFTR induces NF-κB expression and activates transcription of the κB gene. Levels of the inflammatory cytokine ...
The first CF rat model, CFTR tm1sage (termed CFTR-/- or KO), was designed in collaboration with SAGE (now Horizon Discovery) and subsequently characterized at UAB and distributed to the CF community.
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 ...
Eligible patients must have at least one mutation in F508del or another responsive mutation in the CFTR gene. Of note, the CFTR modulator carries a boxed warning over the risk of drug-induced ...
See below for Important Safety Information, including a Boxed Warning. “ALYFTREK is our fifth CFTR modulator to secure FDA approval and represents another significant milestone in our journey to ...