| 摘要 |
A non-human trangenic mammalian animal, as described above, contains an exogenous double stranded DNA sequence stably integratedintot he gemone of the animal, which comprises cis-acting regulatory untis operably linked to a DNA sequence encoding a modifed or variant human FVIII protein, and a signal sequence and secretion motif that is active in directing newly expressed Factor VIII into the milk of the animal at levels an in an unactivated, nondegraded and otherwise stable form that is suitable for subsequent processing for therapeutic applications in treating Hemophilia A. The transgenic mammals are preferably pigs, cows, sheep, goats and rabbits. The applications include milk derivatives used for oral delivery and oral tolerization in the treatment of Hemophilia A.
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