Division of Pneumology, Department of Infectious Diseases and Pneumology, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Gene Ther. 2009 Apr;16(4):574-8. doi: 10.1038/gt.2009.2. Epub 2009 Feb 19.
First-generation adenoviral (Ad) vectors are frequently used vectors for experimental and clinical gene transfer. Earlier it has been shown that parallel overexpression of the cell cycle regulator p21(Waf1/Cip1) (p21) or antiapoptotic bcl-2 from a second vector reduces cytotoxicity and improves transgene expression. Here, we investigate whether the co-expression of p21 and alpha(1)-antitrypsin from a single vector improves vector safety and alpha(1)-antitrypsin expression. Cell lines (A549 and HeLa) and primary cells (small airway epithelial cells and hepatocytes) were infected with adenovirus vectors transducing alpha(1)-antitrypsin with (AdCMV.p21-RSV.hAAT) or without (AdRSV.hAAT) p21. alpha(1)-Antitrypsin expression and cytotoxicity were analyzed using western blot/ELISA and LDH/ALT/AST assays, respectively. Cell cycle profiles were determined by flow cytometry. Co-expression of p21 strongly increased the alpha(1)-antitrypsin expression in all cell types and at all doses tested. No changes in ALT/AST from hepatocytes and only minor increases in the LDH release in A549 and HeLa were observed with either vector. Cell cycle profiles were also not affected adversely. Incorporation of p21 in Ad vectors together with a gene of interest improves the vector performance; such vectors will allow the application of lower doses and thereby reduce immunological side effects.
第一代腺病毒(Ad)载体常用于实验和临床基因转移。早期研究表明,从第二个载体平行过表达细胞周期调节因子 p21(Waf1/Cip1)(p21)或抗凋亡 bcl-2 可以降低细胞毒性并提高转基因表达。在这里,我们研究了从单个载体共表达 p21 和α1-抗胰蛋白酶是否可以提高载体安全性和α1-抗胰蛋白酶表达。细胞系(A549 和 HeLa)和原代细胞(小气道上皮细胞和肝细胞)用转导α1-抗胰蛋白酶的腺病毒载体(AdCMV.p21-RSV.hAAT)或不表达 p21(AdRSV.hAAT)感染。使用 Western blot/ELISA 和 LDH/ALT/AST 测定分别分析α1-抗胰蛋白酶表达和细胞毒性。通过流式细胞术确定细胞周期谱。在所有测试的细胞类型和剂量下,p21 的共表达均强烈增加了α1-抗胰蛋白酶的表达。无论使用哪种载体,来自肝细胞的 ALT/AST 均无变化,A549 和 HeLa 中的 LDH 释放仅略有增加。细胞周期谱也没有受到不利影响。将 p21 纳入腺病毒载体与感兴趣的基因一起可以提高载体性能;这样的载体将允许使用较低的剂量,从而减少免疫副作用。