TRANSGENIC ANIMALS EXPRESSING HUMAN COAGULATION FACTOR VIII AND VON WILLEBRAND FACTOR

摘要

A non-human transgenic mammalian animal contains an exogenous double strandedDNA sequence stably integrated into the genomeof the animal, which comprises cis-acting regulatory units operably linked toa DNA sequence encoding human Factor VIII protein and asignal peptide, where the cis-acting regulatory units are active in mammarygland cells and the signal peptide is active in directing newlyexpressed Factor VIII into the milk of the animal. The promoter may be a milkprotein promoter such as for whey acidic protein, casein,lactalbumin, or beta-lactoglobulin promoter. The transgenic mammals arepreferably farm animals, for example, cows, goats, sheep, rabbitsand pigs. Concurrent expression of a gene for human von Willebrand's Factorinto milk may be used to stabilize newly-secreted FactorVIII.