Cystic Fibrosis

Introduction to Part I: The Relevance of CF Diagnostic Tools for Measuring Restoration of CFTR Function after Therapeutic Interventions in Human Clinical Trials.- High-Throughput Screening of Libraries of Compounds to Identify CFTR Modulators.- Repair of CFTR Folding Defects with Correctors that Function as Pharmacological Chaperones.- Use of Primary Cultures of Human Bronchial Epithelial Cells Isolated from Cystic Fibrosis Patients for the Pre-Clinical Testing of CFTR Modulators.- Design of Gene Therapy Trials in CF Patients.- Nasal Potential Difference Measurements to Assess CFTR Ion Channel Activity.- Measurement of Ion Transport Function in Rectal Biopsies.- Introduction to Part II: RNA Methods to Approach CFTR Expression.- Quantification of CFTR Transcripts.- Nonsense-Mediated mRNA Decay and Cystic Fibrosis.- Approaches to Study CFTR Pre-mRNA Splicing Defects.- Impact of MicroRNA in Normal and Pathological Respiratory Epithelia.- Genomic Approaches to Studying CFTR Transcriptional Regulation.- Introduction to Part III: Biochemical Methods to Study CFTR Protein.- Analysis of CFTR Folding and Degradation in Transiently Transfected Cells.- In Vitro Methods for CFTR Biogenesis.- Analysis of CFTR Interactome in the Macromolecular Complexes.- Methods to Monitor Cell Surface Expression and Endocytic Trafficking of CFTR in Polarized Epithelial Cells.- Segmental and Subcellular Distribution of CFTR in the Kidney.- Endocytic Sorting of CFTR Variants Monitored by Single Cell Fluorescence Ratio Image Analysis (FRIA) in Living Cells.- Introduction to Part IV: Biophysical Methods to Approach CFTR Structure.- CFTR Three-Dimensional Structure.- Molecular Modeling Tools and Approaches for CFTR and Cystic Fibrosis.- Biochemical and Biophysical Approaches to Probe CFTR Structure.- NMR Spectroscopy to Study the Dynamics and Interactions of CFTR.- Introduction to Part V: Assessment of CFTR Function.- Application of High-Resolution Single-Channel Recording to Functional Studies of Cystic Fibrosis Mutants.- Electrophysiological, Biochemical, and Bioinformatic Methods for Studying CFTR Channel Gating and Its Regulation.- CFTR Regulation by Phosphorylation.- How to Measure CFTR-Dependent Bicarbonate Transport: From Single Channels to the Intact Epithelium.