Generation of factor VIII gene knockout mouse by tetraploid embryo complementation technology

Chinese Journal of Medical Genetics

Volumn 27, Issue 1, 2010, Pages 1-6

  Kuang, Ying ^a   Wang, Jin Jin ^b   Lu, Xi Bin ^a   Lu, Shun Yuan ^b   Zhang, Liang Liang ^b   Shen, Chun Ling ^b   Fei, Jian ^a,c   Wang, Zhu Gang ^a,b  

^a Shanghai Research Center for Model Organisms   (China)

^b SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE   (China)

^c TONGJI UNIVERSITY   (China)

Author keywords

Coagulation factor VIII; Gene knockout; Hemophilia A; Tetraploid embryo complementation

Indexed keywords

BLOOD CLOTTING FACTOR 8;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLOOD CLOTTING TIME; CELL CLONE; COMPARATIVE STUDY; CONTROLLED STUDY; EMBRYO; ENZYME ACTIVITY; EXON; GENE; GENE TECHNOLOGY; GENETIC TRANSCRIPTION; HEMOPHILIA A; HETEROZYGOTE; HOMOLOGOUS RECOMBINATION; HOMOZYGOTE; IMMUNOHISTOCHEMISTRY; KNOCKOUT MOUSE; MOUSE; NONHUMAN; PARTIAL THROMBOPLASTIN TIME; PHENOTYPE; POLYMERASE CHAIN REACTION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; TETRAPLOID EMBRYO COMPLEMENTATION TECHNOLOGY;

ANIMALS; DISEASE MODELS, ANIMAL; EMBRYO, MAMMALIAN; FACTOR VIII; FEMALE; HEMOPHILIA A; HUMANS; MALE; MICE; MICE, INBRED ICR; MICE, KNOCKOUT; PARTIAL THROMBOPLASTIN TIME;

MUS;

EID: 76749158991     PISSN: 10039406     EISSN: None     Source Type: Journal    
DOI: 10.3760/cma.j.issn.1003-9406.2010.01.001     Document Type: Article

References (26)

1. Bolton-Maggs PH, Pasi KJ. Haemophilias A and B. Lancet, 2003, 361:1801-1809.
2. Thompson AR. Structure and function of the factor VIII gene and protein. Semin Thromb Hemost, 2003, 29:11-22.
3. Goodeve AC, Peake IR. The molecular basis of hemophilia A: genotype-phenotype relationships and inhibitor development. Semin Thromb Hemost, 2003, 29: 23-30.
4. Andrikovics H, Klein I, Bors A, et al. Analysis of large structural changes of the factor VIII gene, involving intron 1 and 22, in severe hemophilia A. Haematologica, 2003, 88:778-784.
5. Bagnall RD, Waseem N, Green PM, et al. Recurrent inversion breaking intron 1 of the factor VIII gene is a frequent cause of severe hemophilia A. Blood, 2002, 99:168-174.
6. Nakaya SM, Hsu TC, Geraghty SJ, et al. Severe hemophilia A due to a 1.3 kb factor VIII gene deletion including exon 24: homologous recombination between 41 bp within an Alu repeat sequence in introns 23 and 24. J Thromb Haemost, 2004, 2: 1941-1945.
7. Acquila M, Pasino M, Lanza T, et al. Identification of mutations in exon 14 including five novelties in 13 Italian patients with haemophilia A. Haemophilia, 2004, 10:744-746.
8. Rosendaal FR, Smit C, Briet E, et al. Hemophilia treatment in historical perspective: a review of medical and social developments. Ann Hematol, 1991, 62:5.
9. Connelly S, Kaleko M. Gene therapy for hemophilia A. Thromb Haemost, 1997, 78:31.
10. Saenko EL, Ananyeva NM, Moayeri M, et al. Development of improved factor VIII molecules and new gene transfer approachesfor hemophilia A. Curr Gene Ther, 2003, 3:27-41.
11. Balague C, Zhou J, Dai Y, et al. Sustained high level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector. Blood, 2000, 95:820-828.
12. Guo X, Wang H, Chu H, et al. High level expression of human factor VIII in mammalian cells after retroviral-mediated gene transfer. Chin Med J (Engl), 2001, 114:690-693.
13. Kootstra NA, Matsumura R, Verma IM, et al. Efficient production of human F VIII in hemophilic mice using lentiviral vectors. Mol Ther, 2003, 7: 623-631.
14. Tiede A, Eder M, von Depka M, et al. Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction. Gene Ther, 2003, 10: 1917-1925.
15. Moayeri M, Hawley TS, Hawley RG, et al. Correction of murine hemophilia A by hematopoietic stem cell gene therapy. Mol Ther, 2005, 12: 1034-1042.
16. Ye P, Thompson AR, Sarkar R, et al. Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice. Mol Ther, 2004, 10: 117-126.
17. Xu L, Nichols TC, Sarkar R, et al. Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver directed neonatal gene therapy. Proc Natl Acad Sci U S A, 2005, 102: 6080-6085.
18. Hauck B, Xu RR, Xie J, et al. Efficient AAV1-AAV2 hybrid vector for gene therapy of hemophilia. Hum Gene Ther, 2006, 17: 46-54.
19. Xu L, Mei M, Ma X, et al. High expression reduces an antibody response after neonatal gene therapy with B domain-deleted human factor VIII in mice. J Thromb Haemost, 2007, 5: 1805-1812.
20. Matsui H, Shibata M, Brown B, et al. Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors. Stem Cells, 2007, 25, 2660-2669.
21. Ide LM, Gangadharan B, Chiang KY, et al. Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens. Blood, 2007, 110: 2855-2863.
22. Giles AR, Tinlin S, Greenwood R, et al. A canine model of hemophilic (factor VIII: C deficiency) bleeding. Blood, 1982, 60: 727.
23. Bi L, Lawler AM, Antonarakis SE, et al. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet, 1995, 10:119.
24. Bi L, Sarkar R, Naas T, et al. Further characterization of factor VIII-deficient mice created by gene targeting: RNA and protein studies. Blood, 1996, 88: 3446-3450.
25. Yang JL, Gu SP, Chen C, et al. New option for gene-targeting vector construction - Red/ET recombination. Chin J Biotechnol, 2006,22:919-924.
26. Kuang Y, Sun X, Deng T, et al. Production and application of mice completely derived from inbred ES cell lines. Prog Biochem Biophys, 2008,35:304-311.