Description

Part I: Strategies to Correct the Basic Defect in CF and Assess Efficacy in Human Clinical Trials

1. Introduction to Part I: The Relevance of CF Diagnostic Tools for Measuring Restoration of CFTR Function after Therapeutic Interventions in Human Clinical Trials

Kris De Boeck and Melissa Ashlock

2. High-Throughput Screening of Libraries of Compounds to Identify CFTR Modulators

Nicoletta Pedemonte, Olga Zegarra-Moran, and Luis J.V. Galietta

3. Repair of CFTR Folding Defects with Correctors that Function as Pharmacological Chaperones

Tip W. Loo and David M. Clarke

4. Use of Primary Cultures of Human Bronchial Epithelial Cells Isolated from Cystic Fibrosis Patients for the Pre-Clinical Testing of CFTR Modulators

Tim Neuberger, Bill Burton, Heather Clark, and Fredrick Van Goor

5. Design of Gene Therapy Trials in CF Patients

Jane C. Davies and Eric W.F.W. Alton

6. Nasal Potential Difference Measurements to Assess CFTR Ion Channel Activity

Steven M. Rowe, Jean-Paul Clancy, and Michael Wilschanski

7. Measurement of Ion Transport Function in Rectal Biopsies

Martin J. Hug, Nico Derichs, Inez Bronsveld, and Jean Paul Clancy

Part II: RNA Methods to Approach CFTR Expression

8. Introduction to Part II: RNA Methods to Approach CFTR Expression

Ann Harris

9. Quantification of CFTR Transcripts

Anabela S. Ramalho, Luka A. Clarke, and Margarida D. Amaral

10. Nonsense-Mediated mRNA Decay and Cystic Fibrosis

Liat Linde and Batsheva Kerem

11. Approaches to Study CFTR Pre-mRNA Splicing Defects

Elisa Goina, Eugenio Fernandez-Alanis, and Franco Pagani

12. Impact of MicroRNA in Normal and Pathological Respiratory Epithelia

Lisa Giovannini-Chami, Nathalie Grandvaux, Laure-Emmanuelle Zaragosi, Karine Robbe-Sermesant, Brice Marcet, Bruno Cardinaud, Christelle Coraux, Yves Berthiaume, Rainer Waldmann, Bernard Mari, and Pascal Barbry

13. Genomic Approaches to Studying CFTR Transcriptional Regulation

Christopher J. Ott and Ann Harris

Part III: CFTR Protein Biogenesis, Folding, Degradation, and Traffic

14. Introduction to Part III: Biochemical Methods to Study CFTR Protein

Margarida D. Amaral and Gergely L. Lukacs

15. Analysis of CFTR Folding and Degradation in Transiently Transfected Cells

Diane E. Grove, Meredith F. N. Rosser, Richard L. Watkins, and Douglas M. Cyr

16. In Vitro Methods for CFTR Biogenesis

Yoshihiro Matsumura, LeeAnn Rooney, and William R. Skach

17. Analysis of CFTR Interactome in the Macromolecular Complexes

Chunying Li and Anjaparavanda P. Naren

18. Methods to Monitor Cell Surface Expression and Endocytic Trafficking of CFTR in Polarized Epithelial Cells

Jennifer M. Bomberger, William B. Guggino, and Bruce A. Stanton

19. Segmental and Subcellular Distribution of CFTR in the Kidney

Fran ois Jouret, Pierre J. Courtoy, and Olivier Devuyst

20. Endocytic Sorting of CFTR Variants Monitored by Single Cell Fluorescence Ratio Image Analysis (FRIA) in Living Cells

Herve Barri re, Pirjo Apaja, Tsukasa Okiyoneda, and Gergely L. Lukacs

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