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LMB Seminar - How small molecules rescue a folding disease: principles learned from the ion channel CFTR

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Cystic fibrosis is a genetic disorder caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR). CFTR belongs to the ATP -Binding Cassette (ABC) transporter family, but uniquely functions as an ion channel to regulate salt and fluid homeostasis. In this talk, I will discuss the molecular details of how CFTR functions, the structural and functional defects caused by mutations, and how drug molecules act to revert these defects.

This talk is part of the MRC LMB Seminar Series series.

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