Research progress of gene therapy of Wiskott-Aldrich syndrome
10.3760/cma.j.cn101070-20191227-01307
- VernacularTitle:Wiskott-Aldrich综合征基因治疗研究进展
- Author:
Yu TIAN
1
;
Rui ZHANG
;
Tianyou WANG
Author Information
1. 国家儿童医学中心，首都医科大学附属北京儿童医院，北京市儿科研究所血液与肿瘤研究室血液肿瘤中心，北京 100045
- From:
Chinese Journal of Applied Clinical Pediatrics
2020;35(13):1034-1036
- CountryChina
- Language:Chinese
-
Abstract:
Wiskott-Aldrich syndrome (WAS) is also called immunodeficiency syndrome complicated with eczema and thrombocytopenia, which is a rare X-linked primary immunodeficiency disease.Mutations in the WAS gene causes the deficiency of WAS protein, impairing normal functions of T and B lymphocytes, and thereby leading to the clinical manifestations of WAS.Hematopoietic stem cell transplantation (HSCT) is the only effective treatment for WAS patients currently.However, HSCT also has disadvantages of few human leucocyte antigen (HLA) matched donors, the risk of graft versus host disease and immune system disorders.With the development of gene editing technology, multiple studies have established that gene therapy is a promising therapy for WAS.In this study, recent research on the application of gene therapy in WAS were reviewed to promote clinicians′ understanding of gene therapy in the management of WAS.
