NACFC 2020 | S09: CFTR-Directed Therapy Experiences in Animal Models

Published on november 19, 2020 by

This symposium will provide a broad overview of available cystic fibrosis animal models with focus on specific contributions towards addressing unresolved/new challenges for CFTR-directed therapy.

Numerous CF animal models have been developed to study CF pathophysiology and, at least in theory, therapy. Ironically, the best therapies currently available for most CF patients were not developed using CF animal models (e.g. Trikafta). Do animal models still have a place in CF research? And if so, what do they have to offer?

This symposium will provide a review of the current leading CF animal models, and focus on their ability to address unresolved/new challenges for CFTR-directed therapy. Potential CF therapies span a range of agents, delivery routes, and outcome measures, so multiple models could offer different but equally useful testing platforms.

Speakers will also address current and emerging technologies that can be applied to facilitate effectiveness and improve outcome measurement.


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