A factor IX-deficient mouse model for hemophilia B gene therapy
Open Access
- 14 October 1997
- journal article
- research article
- Published by Proceedings of the National Academy of Sciences in Proceedings of the National Academy of Sciences
- Vol. 94 (21) , 11563-11566
- https://doi.org/10.1073/pnas.94.21.11563
Abstract
We have generated a mouse where the clotting factor IX (FIX) gene has been disrupted by homologous recombination. The FIX nullizygous (−/−) mouse was devoid of factor IX antigen in plasma. Consistent with the bleeding disorder, the factor IX coagulant activities for wild-type (+/+), heterozygous (+/−), and homozygous (−/−) mice were 92%, 53%, and FIX gene via adenoviral vectors. Thus, these factor IX-deficient mice provide a useful animal model for gene therapy studies of hemophilia B.Keywords
This publication has 23 references indexed in Scilit:
- Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectorsNature Genetics, 1997
- MHC class I-cestricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenovirusesImmunity, 1994
- In Vivo Gene Therapy of Hemophilia B: Sustained Partial Correction in Factor IX-Deficient DogsScience, 1993
- [6] Human factor IX and factor IXaPublished by Elsevier ,1993
- Targeted mutation of the gene encoding the low affinity NGF receptor p75 leads to deficits in the peripheral sensory nervous systemCell, 1992
- Neonatal lethality and lymphopenia in mice with a homozygous disruption of the c-abl proto-oncogeneCell, 1991
- Deduced amino acid sequence of mouse blood-coagulation factor IXGene, 1990
- An alternative approach to somatic cell gene therapy.Proceedings of the National Academy of Sciences, 1988
- Characteristics of a Human Cell Line Transformed by DNA from Human Adenovirus Type 5Journal of General Virology, 1977
- Christmas DiseaseBMJ, 1952