遗传性肾性尿崩症的药物治疗

Drug treatment of congenital nephrogenic diabetes insipidus

遗传性肾胜尿崩症由精氨酸加压素受体2（AVPR2）或水通道蛋白一2（AQP2）基因突变引起。目前临床治疗主要是改善多饮、多尿的症状。随着对发病机制研究的不断深入，新型药物如AVPR2激动剂和拮抗剂、磷酸二酯酶抑制剂、他汀类、前列腺素E受体激动剂等，通过改善突变的AVPR2／AQP2在细胞内的合成、修饰、表达等达到治疗的目的。

Congenital nephrogenic diabetes insipidus is caused by mutations of Arginine vasopressin receptor 2（AVPR2）/Auqaporin-2（AQP2）. The current clinical treatment is aimed at improving symptoms of polydipsia, polyuria. With the deepening research on the pathogenesis, AVPR2 agonists and antagonists, phosphodiesterase antagonists, statins, prostaglandin E receptor agonists can improve mutational AVPR2/ AQP2 in synthesis, modification, expression in cells, so achieve the goal of treatment.