基因编辑治疗原发性免疫缺陷病

Gene editing for the treatment of primary immunodeficiency disease

摘要:

基因编辑是基于人工核酸酶的先进技术，可对基因组序列进行精确修饰。基因编辑在医药领域展现了巨大的应用前景，为疾病的治疗提供了新的精准治疗方法。原发性免疫缺陷病是单基因突变导致的以反复、难治性感染为标志性表现的一组疾病，病死率极高。而基因编辑的使用给该病带来了治愈的希望。该文综述了基因编辑技术的发展，并简要介绍基因编辑技术在原发性免疫缺陷病中的研究与应用。

Abstract:

Gene editing is an advanced technique based on artificial nucleases and can precisely modify genome sequences. It has shown great application prospects in the field of medicine and has provided a new precision therapy for diseases. Primary immunodeficiency disease is a group of diseases caused by single gene mutation and characterized by recurrent and refractory infections, with an extremely high mortality rate. The application of gene editing has brought hope for curing these diseases. This article reviews the development of gene editing technology and briefly introduces the research and application of gene editing technology in primary immunodeficiency disease.