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Curr Drug Targets. 2019;20(7):748-755. doi: 10.2174/1389450120666190214115309.

Targeting Water in the Brain: Role of Aquaporin-4 in Ischemic Brain Edema.

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Neuroscience and Neuroengineering Research Center, Med-X Research Institute, School of Biomedical Engineering, Shanghai Jiao Tong University, Shanghai 200030, China.
Institute of Molecular Health Sciences, ETH Zurich, 8093 Zurich, Switzerland.
Department of Neurology, Ruijin Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai 200025, China.


Brain edema primarily occurs as a consequence of various cerebral injuries including ischemic stroke. Excessive accumulation of brain water content causes a gradual expansion of brain parenchyma, decreased blood flow and increased intracranial pressure and, ultimately, cerebral herniation and death. Current clinical treatment for ischemic edema is very limited, therefore, it is urgent to develop novel treatment strategies. Mounting evidence has demonstrated that AQP4, a water channel protein, is closely correlated with brain edema and could be an optimal therapeutic target for the reduction of ischemic brain edema. AQP4 is prevalently distributed in the central nervous system, and mainly regulates water flux in brain cells under normal and pathological conditions. This review focuses on the underlying mechanisms of AQP4 related to its dual role in edema formation and elimination.


Aquaporin-4; brain edema; central nervous system; stroke; therapeutic target; water channel.

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