Advances in gene therapy for inborn errors of immunity
10.7499/j.issn.1008-8830.2404027
- VernacularTitle:基因治疗在免疫出生错误中的研究进展
- Author:
Ting LI
1
;
Hong-Mei SONG
Author Information
1. 中国医学科学院北京协和医院儿科,北京 100730
- Keywords:
Inborn error of immunity;
Gene therapy;
Severe combined immunodeficiency;
Wiskott-Aldrich syndrome;
Deficiency of adenosine deaminase 2
- From:
Chinese Journal of Contemporary Pediatrics
2024;26(8):865-870
- CountryChina
- Language:Chinese
-
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.
