[1] These mutations are called gating mutations. The CFTR protein reaches the cell membrane but the channel does not open properly and chloride transport cannot occur (Figure 1d). Class IV ...
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 ...
CF often arises from mutations that make the cystic fibrosis transmembrane conductance regulator (CFTR) protein misfolding and malfunction, causing thick mucus to aggregate in the lungs while also ...
In some people with cystic fibrosis, rare mutations in the CFTR gene cause little-to-no CFTR protein to be produced, meaning there is no protein available to respond to a CFTR modulator therapy.
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.
BOSTON, Nov. 18, 2024 /PRNewswire/ -- Sionna Therapeutics, a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by ...
The drug is expected to reach the more than 5,000 patients who don’t make any cystic fibrosis transmembrane conductance regulator (CFTR) protein, adding to the more than 65,000 people treated ...
by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced the appointment of Laurie Stelzer to the ...