Wu Zhi-Jian, Wu Xiao-Bing, Wang Hong, Lu Bin, Dong Xiao-Yan, Hou Yun-De
State Key Laboratory for Molecular Virology and Genetic Engineering, Institute of Virology, Chinese Academy of Preventive Medicine, Beijing 100052, China.
Sheng Wu Hua Xue Yu Sheng Wu Wu Li Xue Bao (Shanghai). 2002 Mar;34(2):176-80.
In order to achieve sustained human erythropoietin (hEPO) expression in vivo for the treatment of anemias, recombinant adeno-associated virus (rAAV)-mediated hEPO transfer was studied in this report. rAAV vector plasmid carrying hEPO was constructed, and rAAV vector cell line for production of rAAV was also established. By using the one helper virus-one vector cell line strategy, which we reported previously, rAAV containing the hEPO expression cassette was produced in large-scale. The results showed that the hEPO expressed effectively by rAAV-mediated hEPO transfer into cultured BHK-21 cells. Intramuscular injection of rAAV-hEPO to Balb/c mice resulted in the in vivo expression of hEPO for at least ten weeks, along with the significant elevation of the hematocrits. This report indicates the potential use of rAAV-mediated gene transfer for the treatment of various anemias.
为了在体内实现人促红细胞生成素（hEPO）的持续表达以治疗贫血，本报告研究了重组腺相关病毒（rAAV）介导的hEPO转移。构建了携带hEPO的rAAV载体质粒，并建立了用于生产rAAV的rAAV载体细胞系。通过使用我们之前报道的单辅助病毒-单载体细胞系策略，大规模生产了含有hEPO表达盒的rAAV。结果表明，rAAV介导的hEPO转移到培养的BHK-21细胞中后，hEPO能有效表达。向Balb/c小鼠肌肉注射rAAV-hEPO导致hEPO在体内表达至少十周，同时血细胞比容显著升高。本报告表明rAAV介导的基因转移在治疗各种贫血方面具有潜在用途。
