A factor IX-deficient mouse model for hemophilia B gene therapy

被引：138

作者：

Wang, LL

Zoppe, M

Hackeng, TM

Griffin, JH

Lee, KF

Verma, IM

机构：

[1] SALK INST, GENET LAB, SAN DIEGO, CA 92186 USA

[2] SALK INST, CLAYTON FDN LABS PEPTIDE BIOL, SAN DIEGO, CA 92186 USA

[3] Scripps Res Inst, DEPT MOL & EXPT MED, LA JOLLA, CA 92037 USA

[4] Scripps Res Inst, DEPT VASC BIOL, LA JOLLA, CA 92037 USA

来源：

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA | 1997年 / 94卷 / 21期

关键词：

D O I：

10.1073/pnas.94.21.11563

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

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.

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页码：11563 / 11566

页数：4

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