In this session, we examine recent breakthroughs in model systems that enhance our understanding of cystic fibrosis pathogenesis and biology. We explore advancements made in the field of differentiated induced pluripotent stem cells as in vitro models for airway epithelia.
We also highlight progress made in ex vivo model systems that enable the study of large and small human airways including submucosal glands. Advancements in mouse models for studying non-sense CFTR mutation correction are also presented, as well as progress made in ferret models for delivery of gene editing molecules. Lastly, we highlight progress made in cystic fibrosis sheep models and the insight they provide for corrective therapies.
Originally recorded November 4, 2021.
