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.

CF mice in this category have been engineered to express altered CFTR mRNA and protein that correspond to mutations found ... CF null (CFTR-/-) rat: The first CF rat model, CFTRtm1sage, was designed ...

The patient’s cells must produce the CFTR protein for the existing therapies to work ... they injected the lipid nanoparticles intravenously into a cystic fibrosis mouse model harboring the nonsense ...

Sionna Therapeutics recently secured an initial public offering (IPO) of $191m on the Nasdaq stock exchange, signalling ...

In recent years, doctors have started using so-called CFTR modulator therapies to enhance the protein's function ... Scnn1b-transgenic mouse as an animal model for chronic obstructive pulmonary ...

In cystic fibrosis (CF), genetic mutations lead to faulty versions of CFTR, a protein key to moving ions in and out of cells. Improperly functioning CFTR leads to mucus buildup in the lungs of CF ...