UCSF

Key PointsAquaporins (AQPs) are water channel proteins that are expressed in the plasma membranes of cells. They are involved in many physiological functions, including renal water balance, epithelial fluid secretion, cell migration, brain oedema and metabolism in adipocytes.Aquaporinopathies include nephrogenic diabetes insipidus (which is caused by a loss-of-function mutation in AQP2) and neuromyelitis optica (which is caused by the development of AQP4-targeted autoantibodies).Modulators of AQP function are predicted to have broad clinical indications in oedema, cancer, obesity, brain injury, glaucoma, epilepsy and inflammation.The identification of useful small-molecule AQP inhibitors has been slow, in part because of technical challenges in assaying the water-transporting function of AQPs and challenges associated with targeting the compact, pore-containing AQP molecule.