Persistent systemic production of human factor IX in mice by skeletal myoblast-mediated gene transfer: Feasibility of repeat application to obtain therapeutic levels

Blood

Volumn 90, Issue 3, 1997, Pages 1075-1082

  Wang, Jian Min ^a   Zheng, Hong ^a   Blaivas, Mila ^a   Kurachi, Kotoku ^a  

^a UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; ENZYME RELEASE; GENE TRANSFER; HEMOPHILIA B; MALE; MOUSE; MYOBLAST; NONHUMAN; PRIORITY JOURNAL; SCID MOUSE;

EID: 0030795619     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v90.3.1075     Document Type: Article

References (38)

1. Kurachi K, Kurachi S, Furukawa M, Yao SN: Biology of factor IX. Blood Coagul Fibrinolysis 4:953, 1993
2. White GC, Shapiro AD, Ragni MV, Courter SG, Tubridy KL, Kaye JA, Kessler CM: A pharmacokinetic evaluation of recombinant human factor IX (rhFIX) in patients with severe hemophilia B. Thromb Haemost 73:1015, 1995
3. Kurachi K, Wang JM: Gene therapy for hemophilia B, in Lemoine NR, Cooper DN (eds): Gene Therapy. Oxford, UK, Bios Scientific P, 1996, p 213
4. Brownlee GG: Prospects for gene therapy of hemophilia A and B. Br Med Bull 51:91, 1995
5. Kurachi K, Yao SN: Gene therapy by myoblast-mediated gene transfer, in Chang PL (ed): Somatic Gene Therapy. Ann Arbor, MI, CRC Press, 1995, p 121
6. Partridge TA, Davies KE: Myoblast-based gene therapies. Br Med Bull 51:123, 1995
7. Yao SN, Smith KJ, Kurachi K: Primary myoblast-mediated gene transfer: Persistent expression of human factor IX in mice. Gene Ther 1:99, 1994
8. Dai Y, Roman M, Naviaux RK, Verma IM: Gene therapy via primary myoblasts: Long-term expression of factor IX protein following transplantation in vivo. Proc Natl Acad Sci USA 89:10892, 1992
9. 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 262:117, 1993
10. Dai Y, Schwarz EM, Gu D, Zhang WW, Sarvetnick N, Verma IM: Cellular and humoral immune 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
11. 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
12. Yao SN, Farjo A, Roessler BJ, Davidson BL, Kurachi K: Adenovirus-mediated transfer of human factor IX gene in immunodeficient and normal mice: Evidence for prolonged stability and activity of the transgene in liver. Viral Immunol 9:141, 1996
13. Acsadi G, Massie B, Jani A: Adenovirus-mediated gene transfer into striated muscles. J Mol Med 73:165, 1995
14. Walter J, You Q, Hagstrom JN, Sands M, High KA: Successful expression of human factor IX following repeat administration of adenoviral vector in mice. Proc Natl Acad Sci USA 93:3056, 1996
15. 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
16. Yao SN, Kurachi K: Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc Natl Acad Sci USA 89:3357, 1992
17. Donahue RE, Kessler SW, Bodine D, McDonagh K, Dunbar C, Goodman S, Agricola B, Byrne E, Raffeld M, Moen R, Bacher J, Zsebo KM, Nienhuis AW: Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J Exp Med 176:1125, 1992
18. 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
19. Heartlein MW, Roman VA, Jiang JL, Sellers JW, Zuliani AM, Treco DA, Selden RF: Long-term production and delivery of human growth hormone in vivo. Proc Natl Acad Sci USA 91:10967, 1994
20. Dahler A, Wade RP, Muscat GE, Waters MJ: Expression vectors encoding human growth hormone (hGH) controlled by human muscle-specific promoters: Prospects for regulated production of hGH delivered by myoblast transfer or intravenous injection. Gene 145:305, 1994
21. Bansal V, Mowery-Rushton P, Lucht L, Li J, Bahnson A, Watkins SC, Barranger JA: Transduction, expression, and secretion of human glucocerebrosidase by murine myoblasts. Ann NY Acad Sci 716:307, 1994
22. Hamamori Y, Samal B, Tian J, Kedes L: Myoblast transfer of human erythropoietin gene in a mouse model of renal failure. J Clin Invest 95:1808, 1995
23. Wang JM, Zheng H, Sugahara Y, Tan J, Yao SN, Olson E, Kurachi K: Construction of human factor IX expression vectors in retroviral vector frames optimized for muscle cells. Human Gene Ther 7:1743, 1996
24. Price J, Turner D, Cepko C: Lineage analysis in the vertebrate nervous system by retrovirus-mediated gene transfer. Proc Natl Acad Sci USA 84:156, 1987
25. Yao SN, Wilson JM, Nabel EG, Kurachi S, Hachiya HL, 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
26. Southern PJ, Berg P: Transformation of mammalian cells to antibiotic resistance with a bacterial gene under control of the SV40 early region promoter. J Mol Appl Genet 1:327, 1982
27. Herbomel P, Bourachot B, Yaniv M: Two distinct enhancers with different cell specificities coexist in the regulatory region of polyoma. Cell 39:653, 1984
28. Yao SN, Kurachi K: Implanted myoblasts not only fuse with myofibers but also survive as muscle precursor cells. J Cell Sci 105:957, 1993
29. Sheehan DC, Hrapchak BB: Theory and Practice of Histotechnology. St Louis, MO, CV Mosby, 1980
30. Sugahara Y, Catalfamo J, Brooks M, Hitomi E, Bajaj SP, Kurachi K: Isolation and characterization of canine factor IX. Thromb Haemost 75:450, 1996
31. Rando TA, Blau HM: Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy. J Cell Biol 125:1275, 1994
32. Smith KJ, Thompson AR: Labeled factor IX kinetics in patients with hemophilia-B. Blood 58:625, 1981
33. Gussoni E, Pavlath GK, Lanctot AM, Sharma KR, Miller RG, Steinman L, Blau HM: Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation. Nature 356:435, 1992
34. Huard J, Bouchard JP, Roy R, Malouin F, Dansereau G, Labrecque C, Albert N, Richards CL, Lemieux B, Tremblay JP: Human myoblast transplantation: Preliminary results of 4 cases. Muscle Nerve 15:550, 1992
35. Rao SS, Kohtz DS: Positive and negative regulation of D-type cyclin expression in skeletal myoblasts by basic fibroblast growth factor and transforming growth factor beta. A role for cyclin D1 in control of myoblast differentiation. J Biol Chem 270:4093, 1995
36. Brem H, Klagsbrun M: The role of fibroblast growth factors and related oncogenes in tumor growth. Cancer Treat Res 63:211, 1992
37. Kandel J, Bossy-Wetzel E, Radvanyi F, Klagsbrun M, Folk-man J, Hanahan D: Neovascularization is associated with a switch to the export of bFGF in the multistep development of fibrosarcoma. Cell 66:1095, 1991
38. Roman M, Axelrod JH, Dai Y, Naviaux RK, Friedman T, Verma IM: Circulating human or canine factor IX from retrovirally transduced primary myoblasts and established myoblast cell lines grafted into murine skeletal muscle. Somat Cell Mol Gen 18:247, 1992