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CFTR modulation in health and disease

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The cystic fibrosis transmembrane conductance regulator (CFTR) is a unique member of the ATP-binding cassette (ABC) transporter superfamily that forms a gated pathway for the passive movement of anions across cell membranes (see Hwang and Sheppard 2009 for review of its gating properties*). It plays a fundamental role in salt and water transport across epithelia in many tissues, a role that is highlighted by the devastating consequences of mutations, which cause the genetic disease cystic fibrosis. Thus knowledge of CFTR function is crucial to the development of transformational therapies that target the root cause of cystic fibrosis.

* Gating of the CFTR Cl- channel by ATP-driven nucleotide-binding domain dimerisation (Hwang TC, Sheppard DN, 2009)

This area contributes to the wider Cell Signalling and Biology research theme within the School of Physiology and Pharmacology.