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Fixing human factor IX (fIX): correction of a cryptic RNA splice enables the production of biologically active fIX in the mammary gland of transgenic mice.
Yull F, Harold G, Wallace R, Cowper A, Percy J, Cottingham I, Clark AJ
(1995) Proc Natl Acad Sci U S A 92: 10899-903
MeSH Terms: Animals, Animals, Genetically Modified, Base Sequence, Biological Assay, Factor IX, Female, Gene Expression, Genes, Humans, Introns, Mammary Glands, Animal, Mice, Mice, Transgenic, Milk, Molecular Sequence Data, RNA Splicing, RNA, Messenger, Recombinant Proteins
Show Abstract · Added February 19, 2015
Transgenic mice and sheep secrete only low levels of human factor IX in their milk because of an aberrant splicing of the transgene RNA in the mammary gland. Removal of the cryptic 3' splice site prevents this splicing and leads to the production of relatively high levels of factor IX. The purified protein is fully active showing that the mammary gland is capable of the efficient post-translational modification of this protein and that transgenic animals are a suitable means of its production.
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