Research progress of gene therapy of Wiskott-Aldrich syndrome / 中华实用儿科临床杂志

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.