Li Ting, Song Hong-Mei
Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China.
Zhongguo Dang Dai Er Ke Za Zhi. 2024 Aug 15;26(8):865-870. doi: 10.7499/j.issn.1008-8830.2404027.
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.
遗传性免疫缺陷病(IEI)是由免疫系统结构或功能缺陷引起的一组多样的疾病,涉及固有免疫和适应性免疫。2022年IEI分类更新包括485种不同疾病,分为十个主要疾病组。随着分子生物学的快速发展,许多IEI的具体发病机制已被揭示,使得基因治疗在这类疾病的临床前和临床研究中成为可能。本文综述了IEI基因治疗的进展,旨在提高对这些疾病的认识和理解。