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Cystic Fibrosis

Volume 741 of the series Methods in Molecular Biology pp 321-327

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Introduction to Section IV: Biophysical Methods to Approach CFTR Structure

  • Juan L. MendozaAffiliated withDepartment of Physiology, University of Texas Southwestern Medical Center
  • , André SchmidtAffiliated withDepartment of Physiology, University of Texas Southwestern Medical Center
  • , Philip J. ThomasAffiliated withDepartment of Physiology, University of Texas Southwestern Medical Center

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Abstract

Inefficient folding of CFTR into a functional three-dimensional structure is the basic pathophysiologic mechanism leading to most cases of cystic fibrosis. Knowledge of the structure of CFTR and placement of these mutations into a structural context would provide information key for developing targeted therapeutic approaches for cystic fibrosis. As a large polytopic membrane protein containing disordered regions, intact CFTR has been refractory to efforts to solve a high-resolution structure using X-ray crystallography. The following chapters summarize current efforts to circumvent these obstacles by utilizing NMR, electron microscopy, and computational methodologies and by development of experimental models of the relevant domains of CFTR.

Key words

CFTR structure NMR EM crystallography spectroscopy