hEPO Transfer and
Expression by a Recombinant Adeno-associated Virus Vector
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 )
Abstract 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.
Key words recombinant adeno-associated virus; hEPO;
gene transfer; gene therapy; vector
*Corresponding author:
Tel, 86-10-63581302; e-mail, [email protected]