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. |