The main objectives of this project are to identify new chemical compounds, belonging to the family of aminoarylthiazoles (AATs), which have the ability to correct the CFTR protein defect caused by F508del, the most frequent mutation among CF patients.
For the chemical synthesis of aminoarylthiazoles the researchers will use conventional methods of organic synthesis (Hantzsch synthesis). All the molecules will be purified with chromatographyc methods (HPLC) and characterized with mass spectrometry analysis. Using functional and biochemical assays they will test the ability of novel AATs to recover the expression and activity of mutant CFTR. They expect to identify new compounds belonging to the class of aminoarylthiazoles able to stimulate chloride/bicarbonate transport in CF epithelial cells by correction of mutant CFTR-F508del.