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TGN-020 (2-(nicotinamido)-1,3,4-thiadiazole) is a small-molecule research compound originally identified by researchers at Niigata University as an inhibitor of aquaporin-4 (AQP4), the most abundant water channel in the mammalian central nervous system. Discovered via virtual screening based on structural similarities to carbonic anhydrase inhibitors and antiepileptics, TGN-020 has been widely utilized in preclinical research to study brain water homeostasis and glymphatic system function. In animal models, administration of TGN-020 has been shown to reduce brain edema, infarct size, and glial scarring following cerebral ischemia (stroke) or spinal cord compression injury, and to suppress retinal edema in diabetic retinopathy models. Additionally, a carbon-11-labeled version, [11C]TGN-020, has been developed as a positron emission tomography (PET) radioligand for AQP4 imaging. Although its direct channel-blocking mechanism has been disputed in some recent studies, TGN-020 remains a standard pharmacological tool for investigating AQP4-mediated processes.
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