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Publication : A coagulation factor IX-deficient mouse model for human hemophilia B.

First Author  Lin HF Year  1997
Journal  Blood Volume  90
Issue  10 Pages  3962-6
PubMed ID  9354664 Mgi Jnum  J:44497
Mgi Id  MGI:1100381 Doi  10.1182/blood.v90.10.3962
Citation  Lin HF, et al. (1997) A coagulation factor IX-deficient mouse model for human hemophilia B. Blood 90(10):3962-6
abstractText  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 neoDeltaHPRT, 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.
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