QEHB Research Labs, University of Birmingham, Mindelsohn Way, Birmingham, B15 2WB, UK,
BioDrugs. 2013 Dec;27(6):547-58. doi: 10.1007/s40259-013-0042-5.
Alpha 1 antitrypsin deficiency (AATD) is a rare cause of chronic obstructive pulmonary disease. The lung disease is thought to be caused primarily by a lack of effective protection against the harmful effects of neutrophil elastase due to the low AAT levels in the lung. Patients may also develop liver disease due to polymerisation of AAT within hepatocytes. Consequently there has been much research over the years into AAT augmentation therapy in patients with lung disease, initially intravenously, and more recently in inhaled forms. This review article will discuss the role of augmentation therapy in AATD and the current status of recombinant AAT. The potential for other therapeutic strategies, such as blocking polymer formation, enhancing autophagy, gene therapy and stem cell-based treatment, will also be discussed more briefly.
α1-抗胰蛋白酶缺乏症（AATD）是慢性阻塞性肺疾病的一个罕见病因。据认为，肺部疾病主要是由于肺内 AAT 水平低，导致对中性粒细胞弹性蛋白酶的有害作用缺乏有效保护而引起的。由于 AAT 在肝细胞内聚合，患者也可能发生肝病。因此，多年来，人们一直在研究 AAT 对肺病患者的补充治疗，最初是静脉内，最近是吸入形式。本文将讨论补充治疗在 AATD 中的作用以及重组 AAT 的现状。还将更简要地讨论其他治疗策略的潜力，如阻止聚合物形成、增强自噬、基因治疗和基于干细胞的治疗。
