Bellini Maria Helena, Peroni Cibele Nunes, Bartolini Paolo
Biotechnology Department, IPEN-CNEN, University of São Paulo, Brazil.
FASEB J. 2003 Dec;17(15):2322-4. doi: 10.1096/fj.03-0018fje. Epub 2003 Oct 2.
Primary human keratinocytes, stably transduced with the human growth hormone (hGH) gene (under control of the retroviral LTR promoter) and selected via geneticin secreted as much as 7 microg hGH/106 cells/day. Their grafting onto immunodeficient dwarf mice (lit/scid) led to hGH levels in the circulation that did not go below 0.2-0.3 ng/ml during a 12 day period (peak value, 1.5 ng/ml at 4 h). This phenomenon was associated with a body weight increase of the grafted animals (0.060 g/animal/day) significantly higher (P<0.01) than that of controls (0.023 g/animal/day). This is the first report describing successful utilization of immunodeficient dwarf mice (lit/scid) in keratinocyte-based hGH gene therapy.
用人生长激素(hGH)基因(在逆转录病毒LTR启动子控制下)稳定转导并经遗传霉素筛选的原代人角质形成细胞,分泌的hGH可达7微克/10⁶细胞/天。将它们移植到免疫缺陷的侏儒小鼠(lit/scid)身上,导致循环中的hGH水平在12天内不低于0.2 - 0.3纳克/毫升(峰值在4小时时为1.5纳克/毫升)。这种现象与移植动物的体重增加(0.060克/动物/天)相关,显著高于(P<0.01)对照组(0.023克/动物/天)。这是第一篇描述在基于角质形成细胞的hGH基因治疗中成功利用免疫缺陷侏儒小鼠(lit/scid)的报告。