| 摘要 |
Retroviral vectors for directing expression of full length factor VIII in transduced host cells, plasmids encoding the same, and host cells transformed, transfected, or transduced therewith are disclosed. Also disclosed are retroviral particles comprising such retrovital vectors, as are methods for making such particles in suitable packaging cells. Retroviral particles so produced may be amphotropic, ecotropic, polytropic, or xenotropic; alternatively, they may comprise chimeric or hybrid envelope proteins to alter host range. Also described are retrovital particles comprising retroviral vectors for directing full length factor VIII expression which are complement resistant. Pharmaceutical compositions comprising retrovital particles of the invention are also disclosed, as are methods of treating mammals, particularly humans, afflicted with hemophilia. |
