[Lasting expression of hF IX mediated by myoblasts in mice].
OBJECTIVE
To develop experimentally gene therapy for hemophilia B by using C2C12 cells and C3H mice as models.


METHODS
The retroviral vector containing MCK enhancer and hCMV promoter was constructed and was used to transfect PA317 cells. Then the viral particles were used to transfect C2C12 cells and select the G418r clones,which were further confirmed by Southern, Northern, Western blots. And then the C2C12 cells expressing hF IX protein were injected into C3H mice and hF IX expression in vivo was studied.


RESULTS
The expression level of hF IX in vivo was positively correlated with the number of injected cells, and the in vitro expression level of these cell clones. The expression of hF IX could be enhanced and prolonged by immunosuppressive agents.


CONCLUSIONS
Allogeneic myoblasts transplantation leads to immunorejection, but repeated injections at a longer intervals could improve the existing rate of transplanted cells. And this might be helpful to gain insight into the myoblast-mediated gene therapy for hemophilia B patients.
Wang
H
H
Institute of Genetics, Fudan University, Shanghai 200433.
Yang
X
X
Bao
Y
Y
Xue
J
J
D000818:Animals; D002460:Cell Line; D005164:Factor IX / Q000235:genetics*; D015870:Gene Expression / Q000276:immunology; D015316:Genetic Therapy; D002836:Hemophilia B / Q000235:genetics / Q000276:immunology / Q000628:therapy; D006801:Humans; D007166:Immunosuppressive Agents / Q000276:immunology; D051379:Mice; D032446:Myoblasts / Q000276:immunology / Q000378:metabolism*; D013997:Time Factors; D014162:Transfection
D007166:Immunosuppressive Agents; D005164:Factor IX