Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver

Blood

Volumn 91, Issue 12, 1998, Pages 4600-4607

  Nakai, Hiroyuki ^b   Herzog, Roland W ^b   Hagstrom, J Nathan ^b   Walter, Johannes ^b   Kung, Szu Hao ^b   Yang, Edmund Y ^b   Tai, Shing Jen ^b   Iwaki, Yuichi ^b   Kurtzman, Gary J ^b   Fisher, Krishna J ^b   Colosi, Peter ^b   Couto, Linda B ^b   High, Katherine A ^a  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CONTROLLED STUDY; GENE EXPRESSION; GENE TRANSFER; LIVER; MOUSE; NEWBORN; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; VIRUS RECOMBINANT;

EID: 0031838721     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v91.12.4600     Document Type: Article

References (39)

1. High KA, Roberts HR: Factor IX, in High KA, Roberts HR (eds): Molecular Basis of Thrombosis and Hemostasis. New York, NY, Dekker, 1995, p 215
2. Palmer TD, Thompson AR, Miller AD: Production of human factor IX in animals by genetically modified skin fibroblasts: Potential therapy for hemophilia B. Blood 73:438, 1989
3. Yao S-N, Wilson JM, Nabel EG, Kurachi S, Hachiya H, Kurachi K: Expression of human factor IX in rat capillary endothelial cells: Toward somatic gene therapy for hemophilia B. Proc Natl Acad Sci USA 88:8101, 1991
4. Wang J-M, Zheng H, Blaivas M, Kurachi K: Persistent production of human factor IX in mice by skeletal myoblast-mediated gene transfer: Feasibility of repeat application to obtain therapeutic levels. Blood 90:1075, 1997
5. Kay MA, Rothenberg S, Landen CN, Bellinger DA, Leland F, Toman C, Finegold M, Thompson AR, Read MS, Brinkhous KM, Woo SLC: In vivo gene therapy of hemophilia B: Sustained partial correction in factor IX-deficient dogs. Science 262:177, 1993
6. Kay MA, Landen CN, Rothenberg SR, Taylor LA, Leland F, Wiehle S, Fang B, Bellinger D, Finegold M, Thompson AR, Read M, Brinkhous KM, Woo SLC: In vivo hepatic gene therapy: Complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc Natl Acad Sci USA 91:2353, 1994
7. Walter J, You Q, Hagstrom N, Sands M, High KA: Successful expression of human factor IX following repeat administration of an adenoviral vector in mice. Proc Natl Acad Sci USA 93:3056, 1996
8. Yang Y, Ertl HCJ, Wilson JM: MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with El-deleted recombinant adenovirus. Immunity 1:433, 1994
9. Yang Y, Jooss KU, Su Q, Ertl HCJ, Wilson JM: Immune responses to viral antigens versus transgene product in the elimination of recombinant adenovirus-infected hepatocytes in vivo. Gene Ther 3:137, 1996
10. Fisher KJ, Jooss K, Alston J, Haecker SE, High KA, Pathak R, Raper SE, Wilson JM: Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med 3:306, 1997
11. Xiao X, Li J, Samulski RJ: Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector, J Virol 70:8098, 1996
12. Flotte TR, Afione SA, Conrad C, McGrath SA, Solow R, Oka H, Zeitlin PL, Guggino WB, Carter BJ: Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adenoassociated virus vector. Proc Natl Acad Sci USA 90:10613, 1993
13. Ferrari FK, Samulski T, Shenk T, Samulski RJ: Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol 70:327, 1996
14. Fisher KJ, Gao G-P, Weitzman MD, DeMatteo R, Burda JF, Wilson JM: Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol 70:520, 1996
15. Russell DW, Alexander IE, Miller AD: DNA synthesis and topoisomerase inhibitors increase transduction by adeno-associated virus vectors. Proc Natl Acad Sci USA 92:5719, 1995
16. Alexander IA, Russell DW, Miller AD: DNA-damaging agents greatly increase the transduction of nondividing cells by adenoassociated virus vectors. J Virol 68:8282, 1994
17. Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, O'Malley KL, During MJ: Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet 8:148, 1994
18. Koeberl DD, Alexander IE, Halbert CL, Russell DW, Miller AD: Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc Natl Acad Sci USA 94:1426, 1997
19. Snyder RO, Miao CH, Patijn GA, Spratt SK, Danos O, Nagy D, Gown AM, Winther B, Meuse L, Cohen LK, Thompson AR. Kay MA: Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet 16:270, 1997
20. Kurachi S, Hitomi Y, Furukawa M. Kurachi K: Role of intron I in expression of the human factor TX gene. J Biol Chem 270:5276, 1995
21. Skulimowski AW, Samulski RJ: Adeno-associated virus: Integrating vectors for human gene therapy. Method Mol Genet 7:3 1995
22. Herzog RW, Hagstrom JN, Kung S-H, Tai SJ, Wilson JM, Fisher KJ, High KA: Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc Natl Acad Sci USA 94:5804, 1997
23. Fisher KJ, Kelly WM, Burda JF, Wilson JM: A novel adenovirus-adeno-associated virus hybrid vector that displays efficient rescue and delivery of the AAV genome. Hum Gene Ther 7:2079, 1996
24. Uetsuki T, Naito A, Nagata S, Karizo Y: Isolation and characterization of the human chromosomal gene for polypeptide chain elongation factor-1α. J Biol Chem 264:5791, 1989
25. Colosi P, Elliger S, Elliger C, Kurtzman GJ: AAV vectors can be efficiently produced without helper virus. Blood 86;627a, 1995 (abstr, suppl I)
26. Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Byrne BJ: Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 93:14082, 1996
27. Dai Y, Schwarz EM, Gu D, Zhang WW, Sarvetnick N, Verma IM: Cellular and humoral responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression. Proc Natl Acad Sci USA 92:1401, 1995
28. Sambrook J, Fritsch EF, Maniatis T: Molecular Cloning. Cold Spring Harbor, NY, Cold Spring Harbor Laboratory Press, 1989
29. Fair DS, Bahnak BR: Human hepatoma cells secrete single chain factor X, prothrombin and antithrombin III. Blood 64:194, 1984
30. Russell DW, Miller AD, Alexander IE: Adeno-associated virus vectors preferentially transduce cells in S phase. Proc Natl Acad Sci USA 91:8915, 1994
31. Smith TAG. Mehaffey MG, Kayda DB, Saunders JM, Yei S, Trapnell BC, McClelland A, Kaleko M: Adenovims-mediated expression of therapeutic plasma levels of human F.IX in mice. Nat Genet 5:397, 1993
32. Halbert CL, Alexander IA, Wolgamot GM, Miller AD: Adeno-associated virus vectors transduce primary cells much less efficiently than immortalized cells. J Virol 69:1473, 1995
33. Leffert HL, Koch KS, Lad PJ, Skelly H, deHemptinne B: Hepatocyte regeneration, replication and differentiation, in Arias I, Popper H, Schachter D, Shafritz DA (eds): The Liver: Biology and Pathology, New York, NY, Raven, 1982, p 601
34. Armentano D, Zabner J, Sacks C, Sookdeo CC. Smith MP, St George JA, Wadsworth SC, Smith AE, Gregory RJ: Effect of the E4 region on the persistence of transgene expression from adenovirus vectors. J Virol 71:2408, 1997
35. Guo ZS, Wang L-H, Eisensmith RC, Woo SLC: Evaluation of promoter strength for hepatic gene expression in vivo following adenovirus-mediated gene transfer. Gene Ther 3:802, 1996
36. Baskar JF, Smith PP, Nilaver G, Jupp RA, Hofmann S, Peffer NJ, Tenney DJ, Colber-Poley AM, Ghazal P, Nelson JA: The enhancer domain of the human cytomegalovirus major immediate-early promoter determines cell type-specific expression in transgenic mice. J Virol 70:3207, 1996
37. 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA 89:89, 1991
38. Kay MA, Li Q, Liu T-J, Leland F. Toman C, Finegold M, Woo SCL: Hepatic gene therapy: Persistent expression of human alpha-1-antitrypsin in mice after direct gene delivery in vivo. Hum Gene Ther 3:641, 1992
39. Samulski RJ, Chang L-S, Shenk T: Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression. J Virol 63:3822, 1989