摘要 |
<P>PROBLEM TO BE SOLVED: To develop a method for producing natural and modified Factor IX. <P>SOLUTION: Recombinant Factor IX characterized by a high percentage of active protein can be obtained in the milk of transgenic animals incorporating chimeric DNA molecules. Transgenic animals are obtained by introducing DNA encoding the Factor IX into developing embryos so that the foreign DNA is stably incorporated in the DNA of germ line cells of the mature animal. Particularly efficient expression was accomplished by using a chimeric construct comprising a mammary gland-specific promotor, and the Factor IX cDNA lacking the complete or any portion of the 5'-untranslated and 3'-untranslated region, which is substituted with a 5'-end and 3'-end of the mouse whey acidic protein gene. In vitro cell cultures of cells transplanted from the transgenic mammal, methods for producing Factor IX from the cell cultures and methods for treating hemophilia B are also described. <P>COPYRIGHT: (C)2010,JPO&INPIT |