Functional Profiling of CFTR-Directed Therapeutics Using Pediatric Patient-Derived Nasal Epithelial Cell Models
Jeffrey KiHyun Park, Anura Shrivastava, Chengkang Zhang, Brian A. Pollok, Walter E. Finkbeiner, Elizabeth R. Gibb, Ngoc P. Ly, Beate Illek
Abstract
depending on the subject. In addition to characterizing the electrophysiological profile of mutant CFTR activity in cultures for five genotypes, our study exemplifies the promising paradigm of bed-to-bench side cooperation and personalized medicine.
Topics & Concepts
Cystic fibrosisIvacaftorPotentiatorAmilorideCystic fibrosis transmembrane conductance regulatorMedicineMolecular biologyPharmacologyInternal medicineBiologyChemistrySodiumOrganic chemistryCystic Fibrosis Research AdvancesCongenital Ear and Nasal Anomalies