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

点击翻译按钮获取中文摘要

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.