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A Coagulation Factor IX-Deficient Mouse Model for Human Hemophilia B
Hui-Feng Lin,
Nobuyo Maeda,
Oliver Smithies,
David L. Straight, and
Darrel W. Stafford
From the Department of Biology, Department of Pathology and Laboratory Medicine, and Center for Thrombosis and Hemostasis, University of North Carolina at Chapel Hill, Chapel Hill, NC.
Coagulation factor IX deficiency causes hemophilia B in humans. We have used gene targeting to develop a coagulation factor IX-deficient (factor IX-knockout) mouse strain. Mouse embryonic stem (ES) cells were targeted by a socket-containing vector that replaces the promoter through exon 3 of the factor IX gene by neo HPRT, which is a functional neo gene plus a partially deleted hypoxanthine phosphoribosyl transferase minigene. Chimeric mice generated using these socket-containing ES cells transmitted the targeted factor IX gene to their female offspring. Male offspring from these females were characterized and shown to exhibit a phenotype similar to hemophilia B. This factor IX-deficient mouse strain will be useful for studying gene therapy methods and structure-function relationships of recombinant factor IX proteins in vivo.
Blood, Vol. 90 No. 10 (November 15), 1997:
pp. 3962-3966
© 1997 by The American Society of Hematology.
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