Human gene therapy model by induction of stable bone marrow chimerim with regulation of proliferative ability of hematopoietic stem cell

造血干细胞基因治疗是X连锁慢性肉芽肿病的一种治疗方法。然而，由于中性粒细胞的消失和功能重建，长期治疗效果并不令人满意。我们尝试通过转导Lnk基因变体来发展治愈性基因治疗模型，该基因变体促进HSC的适当增殖。我们复制了人类基因治疗的临床问题，制作了一个动物基因治疗模型。但是，Lnk变体的转导或表达的功效的确认是困难的。我们重新检查基因载体的修饰或转导方法。

The gene therapy using hematopoietic stem cells (HSCs) is a curative for X-linked chronic granulomatous disease. However, long-term treatment result is not satisfactory because of disappearance of neutrophils with functional reconstitution. We attempt the development of curative gene therapy model by transductionof Lnk-gene variant which promotes appropriate proliferation into HSCs. We made an animal gene therapy model with reproducing clinical problem of human gene therapy. But, the confirmation of the efficacy of transduction or expression of Lnk variant is difficult. We reexamine the modification of a gene vector or methods for transduction.