肝细胞移植治疗α-1抗胰蛋白酶缺乏症的研究进展

Research progress of hepatocyte transplantation treatment for alpha-1 antitrypsin deficiency

α-1抗胰蛋白酶缺乏症是一种常染色体共显性遗传性疾病,以血液中低水平的α-1抗胰蛋白酶为标志,临床上主要表现为年轻患者的肺气肿和急/慢性肝损伤甚至肝癌等,治疗方法包括对症治疗和α-1抗胰蛋白酶的补充,但无法阻止肝脏纤维化的进展。目前国内已有10余例该疾病的报道,但是对该疾病的认识仍然不足,生物治疗药物空白。现就肝细胞移植治疗该疾病的研究进展进行介绍。

Alpha-1 antitrypsin deficiency is an autosomal codominant genetic disease characterized by low levels of alpha-1 antitrypsin in the blood.Clinically,in young patients,it mainly manifests as emphysema,acute/chronic liver injury and liver cancer.The treatment methods include symptomatic treatment and alpha-1 antitrypsin supplementation.However,the existing treatment cannot prevent the liver fibrosis progression.At present,more than ten cases of the disease have been reported in China,but the understanding of this disease is still indecisive.Moreover,there exists no biotherapy drug for this disorder.This article introduces the research progress of hepatocyte transplantation treatment for this disorder.