Axelrod J H, Read M S, Brinkhous K M, Verma I M
Molecular Biology and Virology Laboratory, Salk Institute, San Diego, CA 92138.
Proc Natl Acad Sci U S A. 1990 Jul;87(13):5173-7. doi: 10.1073/pnas.87.13.5173.
Primary skin fibroblasts from hemophilic dogs were transduced by recombinant retrovirus (LNCdF9L) containing a canine factor IX cDNA. High levels of biologically active canine factor IX (1.0 micrograms per 10(6) cells per 24 hr) were secreted in the medium. The level of factor IX produced increased substantially if the cells were stimulated by basic fibroblast growth factor during infection. Additionally, we also report that endothelial cells transduced by this virus can produce high levels of biologically active factor IX. We propose that skin fibroblasts and endothelial cells from hemophilia B dogs may serve as potential venues for the development and testing of models for treatment of hemophilia B by retrovirally mediated gene replacement therapy.
来自血友病犬的原代表皮成纤维细胞被含有犬因子IX cDNA的重组逆转录病毒（LNCdF9L）转导。培养基中分泌出高水平的具有生物活性的犬因子IX（每24小时每10^6个细胞分泌1.0微克）。如果在感染期间用碱性成纤维细胞生长因子刺激细胞，产生的因子IX水平会大幅增加。此外，我们还报告称，被这种病毒转导的内皮细胞能产生高水平的具有生物活性的因子IX。我们提出，来自B型血友病犬的皮肤成纤维细胞和内皮细胞可能成为通过逆转录病毒介导的基因替代疗法治疗B型血友病的模型开发和测试的潜在场所。
