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高歇病(Gaucher disease)是一种葡萄糖脑苷脂酶基因缺陷的遗传病,由于缺少葡萄糖脑苷酯酶,所以自骨髓衍生的巨噬细胞中有大量葡萄糖脑苷脂聚集。外源性注射此酶或骨髓移植对该病都有效,但各有缺点。造血干细胞基因转移对于许多的影响造血组织的遗传或者获得性疾病的治疗具有很大的潜力。过去,人们曾应用将骨髓细胞与逆转录病毒产生细胞、自体基质或表达SCF的小鼠基质细胞株等共培养的方法成功地将外源基因转入大动物如恒河猴的造血干细胞中。本实验研究如何提高用无共培养细胞的逆转录病毒载体将人葡萄糖脑苷脂酶基因转染造血干细胞的效率进而建立适用于临床的条件。
Gaucher disease is a genetic disease that is defective in the glucocerebrosidase gene and accumulates a large amount of glucocerebrosidase from bone marrow-derived macrophages due to a lack of glucocerebrosidase. Exogenous injection of this enzyme or bone marrow transplantation for the disease are effective, but each has its own drawbacks. Hematopoietic stem cell gene transfer has great potential for the treatment of many hereditary or acquired diseases that affect hematopoietic tissue. In the past, a method of co-culturing bone marrow cells with retrovirus-producing cells, autologous matrices or SCF-expressing mouse stromal cell lines has been successfully used to transfer foreign genes into hematopoietic stem cells of large animals such as rhesus monkeys. This study investigated how to improve the efficiency of transfection of human GLP gene into hematopoietic stem cells using a retroviral vector without co-cultured cells to establish a suitable clinical condition.