儿童慢性肉芽肿病移植及基因治疗研究进展

Advances in transplantation and gene therapy of chronic granulomatous disease in children

慢性肉芽肿疾病是一种罕见的原发性免疫缺陷病,当常规护理和治疗失败时,造血干细胞移植可以是一种治愈方式,年龄越轻,效果越佳。然而,随着病毒载体、核酸内切酶及新的基因编辑技术的出现,基因治疗成为另一种有前途的方法,显示了巨大前景。该文重点综述了近年来移植及基因治疗的相关进展。

Chronic granulomatous disease is a rare primary immune deficiency disease.When routine nursing and treatment fail,he⁃matopoietic stem cell transplantation can be a cure.The earlier the age,the better the effect.However,with the emergence of viral vec⁃tors,endonucleases and new gene editing technologies,gene therapy has become another promising method,showing great prospects.This article focuses on the recent advances in transplantation and gene therapy.