title: A factor IX-deficient mouse model for hemophilia B gene therapy.
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 <5%, respectively, in activated partial thromboplastin time assays. Plasma factor IX activity in the deficient mice (-/-) was restored by introducing wild-type murine FIX gene via adenoviral vectors. Thus, these factor IX-deficient mice provide a useful animal model for gene therapy studies of hemophilia B.
authors.affiliation: Laboratory of Genetics, The Salk Institute, P.O. Box 85800, San Diego, CA 92186-5800, USA.
authors.forename: L
authors.initials: L
authors.lastname: Wang
authors.forename: M
authors.initials: M
authors.lastname: Zoppè
authors.forename: T M
authors.initials: TM
authors.lastname: Hackeng
authors.forename: J H
authors.initials: JH
authors.lastname: Griffin
authors.forename: K F
authors.initials: KF
authors.lastname: Lee
authors.forename: I M
authors.initials: IM
authors.lastname: Verma
mesh_terms: D000256:Adenoviridae; D000818:Animals; D005091:Exons; D005164:Factor IX / Q000096:biosynthesis* / Q000235:genetics*; D005260:Female; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D015698:Genomic Library; D002836:Hemophilia B / Q000235:genetics / Q000628:therapy*; D006579:Heterozygote; D006720:Homozygote; D006801:Humans; D008297:Male; D051379:Mice; D008810:Mice, Inbred C57BL; D008815:Mice, Inbred Strains; D018345:Mice, Knockout; D011995:Recombination, Genetic
chemical_list: D005164:Factor IX