【摘要】 为了研究内含子在活体水平对凝血因子Ⅸ的表达增强作用，首先采用lacZ为报告基因，建立了阳离子脂质体SA介导的活体基因转移方法。将带有凝血因子Ⅸ（hFⅨ）cDNA的质粒pCMVIX和带有hFⅨcDNA及其内含子的小基因（minigene）的质粒pCMVi’Ⅸ用SA脂质体包裹，分别经尾静脉注射到Balb／c鼠体内，pCMVIX注射后未在血浆中检测到hFⅨ蛋白；而pCMVi’Ⅸ注射后在Balb／c鼠血浆中检测到hFⅨ蛋白，最高达到10．1ng／ml，持续28天。pCMVi’Ⅸ经二次注射，FⅨ表达水平最高达到43.9ng／ml，持续40天后仍可检测到hFⅨ基因的表达，结果表明，内含子在活体水平对凝血因子Ⅸ的表达有显著的增强作用。更多还原

【Abstract】 In order to study the role of nitron in the expression of human clotting factor Ⅸ in vivo, the gene delivery system mediated by SA liposome are carried out by lac Z reporter gene. Plasmid pCMVIX containing hF Ⅸ cDNA and pCMVi’ Ⅸ containing hF Ⅸ minigene (hF Ⅸ cDNA and part of nitron 1 from hF Ⅸ gene) are mixed with SA liposome and injected into Balb/c mice via tail vein. hF Ⅸ could not be detected in mice injected with pCMV Ⅸ by ELISA. In mice injected with pCMVi’ Ⅸ, the maximum level of hF Ⅸ has reached 10. lug/ml and lasted for 28 days after injection. Follow the second injection of pCMVi’ Ⅸ, the maximum level of hF Ⅸ has reached 43. 9ng/ml and lasted for more than 40 days. The results show that nitron play an important role in the expression of human clotting factor Ⅸ in vovo.更多还原