基因治疗在免疫出生错误中的研究进展

Advances in gene therapy for inborn errors of immunity

摘要:

免疫出生错误（inborn errors of immunity, IEI）是由遗传因素导致免疫结构或功能障碍所致的一类疾病，可累及固有免疫和适应性免疫。2022年IEI新分类包含485种IEI，分为十大类疾病。近年来随着分子生物学的快速发展，许多IEI的具体发病机制得以揭示，使得基因治疗在该类疾病的临床前和临床研究成为可能。该文综述基因治疗在IEI中的研究和应用，以进一步提高临床医生对IEI诊治的认知。

Abstract:

Inborn errors of immunity (IEI) are a diverse group of disorders caused by defects in immune system structure or function, involving both innate and adaptive immunity. The 2022 update of the IEI classification includes 485 distinct disorders, categorized into ten major disease groups. With the rapid development of molecular biology, the specific pathogenesis of many IEI has been revealed, making gene therapy possible in preclinical and clinical research of this type of disease. This article reviews the advancements in gene therapy for IEI, aiming to increase awareness and understanding of these disorders.