Cystic Fibrosis : Diagnosis and Protocols, Volume I: Approaches to Study and Correct CFTR Defects - Amaral, Margarida D.; Kunzelmann, Karl.; SpringerLink (Online service) (Totowa, NJ : Humana Press : Imprint: Humana, 2011)
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.
Keywords: Molecular biology; Human genetics; Laboratory medicine; Molecular Medicine; Human Genetics; Laboratory Medicine