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Nat Rev Drug Discov. 2014 Apr;13(4):259-77. doi: 10.1038/nrd4226. Epub 2014 Mar 14.

Aquaporins: important but elusive drug targets.

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Departments of Medicine and Physiology, 1246 Health Sciences East Tower, University of California, San Francisco, California 94143-0521, USA.
Department of Chemistry and Biochemistry, San Francisco State University, 1600 Holloway Ave, San Francisco, California 94132, USA.
Academic Neurosurgery Unit, Room 1.122 Jenner Wing, St George's, University of London, Tooting, London SW17 0RE, UK.


The aquaporins (AQPs) are a family of small, integral membrane proteins that facilitate water transport across the plasma membranes of cells in response to osmotic gradients. Data from knockout mice support the involvement of AQPs in epithelial fluid secretion, cell migration, brain oedema and adipocyte metabolism, which suggests that modulation of AQP function or expression could have therapeutic potential in oedema, cancer, obesity, brain injury, glaucoma and several other conditions. Moreover, loss-of-function mutations in human AQPs cause congenital cataracts (AQP0) and nephrogenic diabetes insipidus (AQP2), and autoantibodies against AQP4 cause the autoimmune demyelinating disease neuromyelitis optica. Although some potential AQP modulators have been identified, challenges associated with the development of better modulators include the druggability of the target and the suitability of the assay methods used to identify modulators.

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