在目前进行基因治疗的研究中,有可能选择逆转录病毒首先用于人体,就是将克隆的基因输送至骨髓细胞内。对于一些由于骨髓细胞缺乏某种基因,不能产生某种特异性酶的疾病将开展研究。在每种疾病中,酶的产生并不需要过细地调节;在任何水平下如能保持稳定的产生,都能改善病人的状况。最有可能选作治疗用的三种酶是次黄嘌呤-鸟嘌呤磷酸核糖基转移酶、嘌呤核苷酸磷酸化酶和腺苷脱氨酶。缺乏上述第一种酶可导致Lesch-Nyhan综合征,缺乏其余任何一种酶均可引起严重的免疫缺损病。基因的传送有两个步骤。首先,将克隆的基因放入在培养中生长的有核的骨髓干细胞群中,然后将这些细胞移入病人骨髓中增殖,最 In the current study of gene therapy, it is possible to choose the first retrovirus for the human body, that is, the cloned gene delivery to bone marrow cells. For some lack of a certain gene due to bone marrow cells, can not produce a specific enzyme disease will carry out research. In each disease, the production of enzymes does not need to be finely tuned; maintaining stable production at any level can improve the patient’s condition. The three most likely candidates for therapy are hypoxanthine-guanine phosphoribosyltransferase, purine nucleotide phosphorylase and adenosine deaminase. The lack of the first enzyme can lead to Lesch-Nyhan syndrome, the lack of any other enzyme can cause severe immunodeficiency disease. There are two steps to gene delivery. First, the cloned gene is placed in a population of nucleated bone marrow stem cells that grow in culture, and these cells are then transferred into the patient’s bone marrow for proliferation