Methods in Molecular Biology Volume 741, 2011, pp 347-363

Molecular Modeling Tools and Approaches for CFTR and Cystic Fibrosis

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Abstract

Cystic fibrosis is a multi-faceted disease resulting from the dysfunction of the CFTR channel. Understanding the structural basis of channel function and the structural origin of the defect is imperative in the development of therapeutic strategies. Here, we describe molecular modeling tools that, in conjunction with complementary experimental tools, lead to significant findings on CFTR channel function and on the effect of the pathogenic mutant F508del.