This workshop will focus on CFTR biology, from molecular structure/function to impact in disease. The session will introduce the earliest known CFTR by describing its structure and function. It will then be presented how proteostasis network profiling can help predict response to correctors. Additionally, there will be description of how the rate of translation of NBD1 can cause structural changes and affect function of mature CFTR protein. We will also learn about how CF airway epithelial cells exhibit altered localization of epithelial markers leading to a less differentiated and more proliferative state. Finally, a human pancreas-on-a-chip model will be presented as a way to study CF-related diabetes (CFRD).
Define how sequence evolution and protein translation can affect CFTR function.
Evaluate new proteostasis profiling to predict response of CFTR mutants.
Assess the effect of CFTR on new considerations related to epithelial cell differentiation and models of CF-related diabetes.