PTAC/CFTR
David Sheppard
School of Physiology, Pharmacology and Neuroscience, University of Bristol
Christine Bear, PhD
Molecular Medicine
The Hospital for Sick Children
This symposium will discuss new knowledge about the structure of CFTR, its function as a regulated anion channel and the action of CFTR modulators. First, it will reveal how the integration of structural and functional studies reveals the mechanism of action of a CFTR potentiator and a stabilising nanobody. Second, it will highlight the role of protein and lipid interactions in the formation of CFTR-containing clusters in biological membranes and how these interactions might be targeted to develop new therapeutics for cystic fibrosis. Third, it will demonstrate how chemical space can be exploited to identify innovative CFTR modulators that function as both correctors and potentiators. Together, these presentations will inform new research to optimise personalised therapies for people with cystic fibrosis.