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AAV2-hAQP1 is an investigational adeno-associated virus serotype 2 (AAV2) vector-based gene therapy designed to treat radiation-induced xerostomia (dry mouth) and parotid gland hypofunction. Developed by MeiraGTx, the therapy involves the delivery of the human aquaporin-1 (hAQP1) gene directly into the parotid glands. In patients who have undergone radiation therapy for head and neck cancer, the fluid-secreting acinar cells are often destroyed, while ductal cells remain. AAV2-hAQP1 works by transducing these surviving ductal cells, causing them to express the AQP1 water channel protein. This converts the normally water-impermeable ductal cells into water-permeable cells, facilitating the movement of water into the salivary ducts and restoring saliva flow.
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