Improved fluorescence assays to measure the defects associated with F508del-CFTR allow identification of new active compounds

What is it about?

Cystic fibrosis (CF) is a common genetic disease caused by defects in CFTR, a protein that controls fluid movement across the lining of airways and guts. We have developed simple tests that can be used to quantify the CFTR defects most commonly found in patients. To demonstrate the tests' use we screened a set of chemicals and identified a new molecule that can reduce the severity of CFTR malfunction.

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Why is it important?

With the new tests information on CFTR can be obtained relatively easily. This could be valuable when looking for new drugs. In addition, the new molecule has some advantages compared to the drug ivacaftor, currently used by some CF patients, and could be a tool for developing improved drugs.