Whitehead生物医学研究所和Massach-usetts技术研究所的人员在基因治疗上采取了一重要步骤,即将复将的正演常人基因转移到病兔肝细胞培养中,治疗细胞的遗传性缺陷. 转移法使用基因修饰的逆转录病毒,病毒携有为产生肝细胞表面上脂蛋白受体而编码的基因。转化细胞产生的受体为正常肝细胞上受体的5倍。研究者希望使用这种方法治疗家族性血胆固醇过高症。这是一种罕见的病,因遗传缺陷而妨碍了细胞清除血液中低密度脂蛋白 Whitehead Institute of Biomedical Research and Massachusetts Institute of Technology personnel took an important step in gene therapy, that is, the forthcoming forward normal gene transfer to the rabbit liver cell culture, the treatment of genetic defects in cells. Using genetically modified retroviruses, the virus carries genes that are encoded for the production of lipoprotein receptors on the surface of hepatocytes. Transformed cells produce 5 times more receptors on normal liver cells. Researchers hope to use this method to treat familial hypercholesterolemia. This is a rare disease that prevents cells from clearing low-density lipoproteins in the blood due to genetic defects