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Target intelligence / Profile preview
Aquaporin-4 (AQP4)
- Target
- AQP4
- Molecular classification
- Aquaporin, Integral membrane protein, Water channel
01
Overview
Aquaporin-4 (AQP4) is a water channel protein primarily expressed in astrocytes of the central nervous system. It facilitates rapid, selective transport of water across cell membranes and plays a critical role in maintaining CNS water homeostasis, edema formation/resolution, potassium buffering, and cerebrospinal fluid circulation. AQP4 is targeted by autoantibodies in neuromyelitis optica spectrum disorder (NMOSD), leading to inflammatory demyelination. It is also implicated in brain edema following injury or stroke. Potential roles are being investigated for epilepsy, glial scarring, neurodegenerative diseases, and psychiatric disorders.
02
Mechanism of action
Blocks pathogenic antibody binding in NMOSD (aquaporumab)
03
Biological functions
Water transportWater homeostasisPotassium bufferingRegulation of extracellular space volumeCerebrospinal fluid circulationInterstitial fluid movement (glymphatic system)Cell migrationMetabolismSynaptic plasticityMemory formationOsmotic balance
04
Disease associations
Neuromyelitis optica spectrum disorder (NMOSD)Brain edemaCerebral swellingStrokeEpilepsyGlial scarringNeurodegenerative diseasesPsychiatric disorders
05
Safety considerations
Druggability issues with AQPsBroad tissue distribution/physiological importance potentially leading to off-target effects
06
Interacting drugs
Aquaporumab
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