This session will focus on new animal and cellular models that have been developed to further our knowledge of the pathogenesis and treatment of CF disease. New animal models to be discussed include transgenic mice expressing human CFTR mutations created by CRISPR/Cas9 gene editing and transgenic rabbits with gene-targeted knockout of rabbit CFTR. New cellular models to be discussed include human intestinal organoids used to evaluate patient-specific response to CFTR modulators and relations with disease severity and induced-pluripotent stem cells that are differentiated to produce mature human airway epithelium for regenerative medicine purposes. The utility and limitations of these models for understanding CF disease and its treatment will be discussed.
Discuss the contributions of the CF rabbit model and mouse Cftr knockout models that express human CFTR mutations to our understanding of CF airway and intestinal disease including their utility for testing new correctors and potentiators of CFTR.
Explain how human intestinal organoids can be used to evaluate patient-specific response to CFTR modulator treatment and the relationship to disease severity.
Discuss new methods for using iPSC differentiation of lung epithelial cells to develop mature human airway epithelium for transplantation.