APPTT
PTAC/CFTR
Kathleen Boyne, MD
Pediatrics, Division of Pulmonary and Sleep Medicine
Ann & Robert H. Lurie Children's Hospital, Northwestern University Feinberg School of Medicine
Bruce Schultz, PhD
Kansas State University
This workshop will feature new and adapted in vivo models critical for pre-clinical evaluation of therapeutics and proof-of-concept trials in CF. This includes novel models that help advance our understanding of the broader, longitudinal impact of lost CFTR function on multiple cell types and organ systems. Our presenters will review transcriptional profiles of pulmonary ionocytes in the ferret airway in addition to a transgenic murine model with tissue specific CFTR loss of function in smooth muscle. This session will also highlight evaluation of an inhaled "mutation-agnostic" CFTR corrective therapy in non-human primates. Progress using a humanized CF rat model to investigate adjunctive treatments in the setting of modulators will be described. And, finally, we will see how breakthroughs in ferrets with lung disease afford the ability to longitudinally evaluate colonization and the airway microbiome, providing additional opportunity for pre-clinical, proof-of-concept trials.
Speaker: Feng Yuan – University of Iowa
Speaker: Elizabeth Kramer, MD, PhD – Cincinnati Children's Hospital
Speaker: Trevor Parry, PhD – Krystal Biotech, Inc.
Speaker: Lawrence Rasmussen, PhD – University of Alabama at Birmingham
Speaker: Allison Lacina (she/her/hers) – Anatomy & Cell Biology, U of Iowa