Circulating human or canine factor IX from retrovirally transduced primary myoblasts and established myoblast cell lines grafted into murine skeletal muscle.

摘要: We have used retroviral vectors to introduce human or canine factor IX cDNAs into cultured primary murine and myoblasts the established myoblast cell line C2C12. In all cases, stably infected cells produced biologically active in culture secreted detectable amounts medium both before after differentiation of myotubes. Myoblasts differentiated myotubes are therefore capable performing posttranslational modifications coagulation required for biological activity. grafted genetically modified skeletal muscles nude mice detected stable levels circulating up two months grafting. propose that grafting lines muscle may represent a useful approach gene therapy variety genetic diseases, including intrinsic disease defects proteins as hemophilias.