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
Proc Natl Acad Sci U S A. 1995 92 (24): 10899-903

PMID: 7479906 · PMCID: PMC40538 · DOI:10.1073/pnas.92.24.10899

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.

MeSH Terms (18)

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 Recombinant Proteins RNA, Messenger RNA Splicing

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