Grant: Emerging Team Grant: From Genes to Proteins, Cells, Tissues and Patients - LOI

Most cystic fibrosis (CF) is caused by mutations that cause partial misfolding and retention of the cystic fibrosis transmembrane conductance regulator (CFTR) in the endoplasmic reticulum (ER). Despite great strides in basic CF research, there remains no cure. We will determine the mechanisms responsible for CFTR retention in the ER, identify potential targets for small molecules that rescue trafficking of the major disease-causing mutant, establish functional assays and use them to evaluate molecular targets and correctors, and develop new research tools and therapeutic strategies that may be applicable to protein misfolding diseases generally. The work will be carried out by investigators with broad expertise in chemical and structural biology, protein folding, live cell imaging of protein dynamics, CF pathophysiology, and drug discovery.