WT1基因编码一种双向转录因子,对人类泌尿生殖系统的发育和wilms’瘤的发生发展有重要意义。WT1基因也参与人类的造血调控,参与造血细胞增殖、分化、凋亡等各个过程及白血病的形成,在多种白血病中均有异常高表达,故被认为是一种新的白血病抗原,用于制备白血病疫苗、表达WT1白血病细胞的体外净化及体内抗原特异性治疗,近年来利用反义RNA技术进行反义基因治疗也有较快发展。WT1成为白血病基因治疗和特异性免疫治疗的新靶点。 WT1 gene encodes a bidirectional transcription factor, which is of great significance to the development of human urogenital system and the occurrence and development of wilms’ tumor. WT1 is also involved in the regulation of hematopoiesis in humans. It is involved in various processes such as proliferation, differentiation and apoptosis of hematopoietic cells and the formation of leukemia. WT1 gene is highly expressed in many leukemias and is therefore considered as a new leukemia antigen for Preparation of leukemia vaccine, WT1 leukemia cells in vitro purification and in vivo antigen-specific treatment, antisense RNA technology using antisense RNA technology in recent years have also developed rapidly. WT1 has become a new target for leukemia gene therapy and specific immunotherapy.