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

From the reviews:"Summarize the current complex information on cystic fibrosis (CF) and the innovative new technologies available to basic scientists involved in the study of CF. ... This detailed book is aimed at basic research scientists and academicians working on CF. The protocols would be of use to graduate students and postdoctoral fellows as well. ... technology described would be invaluable to clinical laboratories involved in the diagnosis of CF. This represents a must-have guide for research laboratories working on the functional mechanisms of the CFTR gene." (Luis F. Escobar, Doody's Book Reviews, March, 2012)