[hEPO transfer and expression by a recombinant adeno-associated virus vector].
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.
Wu
Zhi-Jian
ZJ
State Key Laboratory for Molecular Virology and Genetic Engineering, Institute of Virology, Chinese Academy of Preventive Medicine, Beijing 100052, China. wuxb0168@sina.com
Wu
Xiao-Bing
XB
Wang
Hong
H
Lu
Bin
B
Dong
Xiao-Yan
XY
Hou
Yun-De
YD
D000818:Animals; D002460:Cell Line; D000229:Dependovirus / Q000235:genetics*; D004921:Erythropoietin / Q000097:blood / Q000235:genetics* / Q000502:physiology; D015870:Gene Expression; D005822:Genetic Vectors / Q000235:genetics; D049452:Green Fluorescent Proteins; D006400:Hematocrit; D006801:Humans; D007273:Injections, Intramuscular; D008164:Luminescent Proteins / Q000235:genetics / Q000378:metabolism; D008297:Male; D051379:Mice; D008807:Mice, Inbred BALB C; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage / Q000235:genetics / Q000378:metabolism; D013997:Time Factors; D014162:Transfection
D008164:Luminescent Proteins; D011993:Recombinant Fusion Proteins; D004921:Erythropoietin; D049452:Green Fluorescent Proteins