α1-抗胰蛋白酶相关疾病及其临床应用进展  

Research progress on related diseases and clinical application of α1-antitrypsin

作　　者：张剑平 周兰贞 郭采平 

机构地区：[1]深圳市卫光生物制品股份有限公司,广东深圳518107

出　　处：《中国输血杂志》2017年第3期316-319,共4页Chinese Journal of Blood Transfusion

基　　金：深圳重组血浆蛋白类创新药物工程实验室项目

摘　　要：α1-抗胰蛋白酶(AAT),是人类血浆中重要的蛋白酶抑制剂,保护机体正常细胞和器官不受蛋白酶的损伤。α1-抗胰蛋白酶缺乏症(AATD)是常见的1种单基因遗传病,患者会出现肺气肿、肝硬化以及其他疾病,严重者甚至出现死亡。α1-抗胰蛋白酶缺乏症常用的治疗方法是静脉输注AAT制剂进行补充治疗,近年来AAT吸入疗法、基因疗法和干细胞疗法取得了新的进展。本文简述了AAT的分子特性、作用机制以及其相关疾病研究进展,并综述了AAT在AATD相关疾病中的应用情况,进而展望了α1-抗胰蛋白酶产品的市场应用及技术发展前景。α1-antitrypsin （AAT）,an human plasma protease inhibitor,plays an important role in protecting the normal cells and organs from protease damage.α1-antitrypsin deficiency （AATD） is a common single-gene disease.The patients with AATD may develop emphysema,cirrhosis and other diseases,even are at risk of death.AAT replacement therapy with intravenous infusions of purified AAT from human plasma is the most usual clinical treatment method for AATD to elevate the serum AAT level and partially recover the biochemical defect.In recent years,several new therapeutic methods for AATD,including the inhaled AAT,gene therapy and stem cell therapy,have been developed.In the present paper,the molecular characteristics,the functional mechanism of AAT and its related diseases were described.In addition,the application of AAT in the AATD-related diseases was reviewed,and the technological development and market prospects of AAT product were studied.

关 键 词：Α1-抗胰蛋白酶 α1-抗胰蛋白酶缺乏症(AATD) 临床应用 研究进展 

分 类 号：R457[医药卫生—治疗学]