Construction of human factor IX expression vectors in retroviral vector frames optimized for muscle cells

Human Gene Therapy

Volumn 7, Issue 14, 1996, Pages 1743-1756

  Wang, Jian Min ^a   Zheng, Hong ^a   Sugahara, Yuichi ^a   Tan, Jun ^a   Yao, Shou Nan ^a   Olson, Eric ^b   Kurachi, Kotoku ^a  

^a UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF TEXAS MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA ACTIN; BETA ACTIN; BLOOD CLOTTING FACTOR 9; CREATINE KINASE; CYTOCHROME C OXIDASE; MYOGENIN;

ANIMAL CELL; ARTICLE; CONTROLLED STUDY; DNA TRANSFECTION; ENHANCER REGION; EXPRESSION VECTOR; GENE TARGETING; GENE THERAPY; HEMOPHILIA B; INTRON; LONG TERMINAL REPEAT; MOUSE; MURINE LEUKEMIA VIRUS; MUSCLE CELL; MYOBLAST; MYOTUBE; NONHUMAN; NORTHERN BLOTTING; NUCLEOTIDE SEQUENCE; POLYADENYLATION; PROMOTER REGION; PROTEIN EXPRESSION; RETROVIRUS; ROUS SARCOMA ONCOVIRUS; SKELETAL MUSCLE;

ANIMALIA; MOLONEY MURINE LEUKEMIA VIRUS; MURINAE; ONCOVIRINAE; RICE STRIPE VIRUS; ROUS SARCOMA VIRUS; UNIDENTIFIED RETROVIRUS;

EID: 0029818271     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1996.7.14-1743     Document Type: Article

References (48)

1. ACSADI, G., MASSIE, B., and JANI, A. (1995). Adenovirus-mediated gene transfer into striated muscles. J. Mol. Med. 73, 165-180.
2. ANSON, D.S., and OCCHIODORO, T. (1994). Transcriptional activity of the CD45 gene promoter in retroviral vector constructs. Biochim. Biophys. Acta 1219, 81-88.
3. BANSAL, V., MOWERY-RUSHTON, P., LUCHT, L., LI, J., BAHNSON, A., WATKINS, S.C., and BARRANGER, J.A. (1994). Transduction, expression, and secretion of human glucocerebrosidase by murine myoblasts. Ann. N.Y. Acad. Sci. 716, 307-319.
4. BAQUE, S., NEWGARD, C.B., GERARD, R.D., GUINOVART, J.J., and GOMEZ-FOIX, A.M. (1994). Adenovirus-mediated delivery into myocytes of muscle glycogen phosphorylase, the enzyme deficient in patients with glycogen-storage disease type V. Biochem. J. 304 (pt. 3), 1009-1014.
5. BARR, E., and LEIDEN, J.M. (1991). Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254, 1507-1509.
6. CONE, R.D., WEBER-BENAROUS, A., BAORTO, D., and MULLIGAN, R.C. (1987). Regulated expression of a complete human β-globin gene encoded by a transmissible retrovirus vector. Mol. Cell Biol. 7, 887-897.
7. DAHLER, A., WADE, R.P., MUSCAT, G.E., and WATERS, M.J. (1994). 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, 305-310.
8. DAI, Y., ROMAN, M., NAVIAUX, R.K., and VERMA, I.M. (1992). Gene therapy via primary myoblasts: Long-term expression of factor IX protein following transplantation in vivo. Proc. Natl. Acad. Sci. USA 89, 10892-10895.
9. DAI, Y., SCHWARZ, E.M., GU, D., ZHANG, W.W., SARVETNICK, N., and VERMA, I.M. (1995). 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, 1401-1405.
10. DAVIS, H.L., DEMENEIX, B.A., QUANTIN, B., COULOMBE, J., and WHALEN, R.G. (1993a). Plasmid DNA is superior to viral vectors for direct gene transfer into adult mouse skeletal muscle. Hum. Gene Ther. 4, 733-740.
11. DAVIS, H.L., WHALEN, R.G., and DEMENEIX, B.A. (1993b). Direct gene transfer into skeletal muscle in vivo: Factors affecting efficiency of transfer and stability of expression. Hum. Gene Ther. 4, 151-159.
12. DHAWAN, J., PAN, L.C., PAVLATH, G.K., TRAVIS, M.A., LANCTOT, A.M., and BLAU, H.M. (1991). Systemic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 254, 1509-1512.
13. DRANOFF, G., JAFFEE, E., LAZENBY, A., GOLUMBEK, P., LEVITSKY, H., BROSE, K., JACKSON, V., HAMAKA, H., PARDOLL, D., and MULLIGAN, R.C. (1993). Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long-lasting anti-tumor immunity. Proc. Natl. Acad. Sci. USA 90, 3539-3543.
14. DWARKI, V.J., BELLONI, P., NIJJAR, T., SMITH, J., COUTO, L., RABIER, M., CLIFT, S., BERNS, A., and COHEN, L.K. (1995). Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc. Natl. Acad. Sci. USA 92, 1023-1027.
15. EDMONDSON, D.G., CHENG, T.C., CSERJESI, P., CHAKRABORTY, T., and OLSON, E.N. (1992). Analysis of the myogenin promoter reveals an indirect pathway for positive auto regulation mediated by the muscle-specific enhancer factor MEF-2. Mol. Cell. Biol. 12, 3665-3677.
16. EUSTICE, D.C., FELDMAN, P.A., COLBERG-POLEY, A.M., BUCKERY, R.M., and NEUBAUER, R.H. (1991). A sensitive method for the detection of β-galactosidase in transfected mammalian cells. BioTechniques 11, 739-742.
17. GROSSMAN, M., RAPER, S.E., KOZARSKY, K., STEIN, E.A., ENGELHARDT, J.F., MULLER, D., LUPIEN, P.J., and WILSON, J.M. (1994). Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia. Nat. Genet. 6, 335-341.
18. HAMAMORI, Y., SAMAL, B., TIAN, J., and KEDES, L. (1995). Myoblast transfer of human erythropoietin gene in a mouse model of renal failure. J. Clin. Invest 95, 1808-1813.
19. HERBOMEL, P., BOURACHOT, B., and YANIV, M. (1984). Two distinct enhancers with different cell specificities coexist in the regulatory region of polyoma. Cell 39, 653-662.
20. JIAO, S., GUREVICH, V., and WOLFF, J.A. (1993). Long-term correction of rat model of Parkinson's disease by gene therapy. Nature 362, 450-453.
21. JONSSON, J.J., HABEL, D.E., and McIVOR, R.S. (1995). Retrovirus-mediated transduction of an engineered intron-containing purine nucleoside phosphorylase gene. Hum. Gene Ther. 6, 611-623.
22. KOST, T.A., THEODORAKIS, N., and HUGHES, S.H. (1983). The nucleotide sequence of the chick cytoplasmic β-actin gene. Nucleic Acids Res. 11, 8287-8301.
23. KURACHI, S., HITOMI, Y., FURUKAWA, M., and KURACHI, K. (1995). Role of intron I in expression of the human factor IX gene. J. Biol. Chem. 270, 5276-5281.
24. LOMAX, M.I., RIGGS, P.K., and WOMACK, J.E. (1995). Structure and chromosomal location of the bovine gene for the heart muscle isoform of cytochrome c oxidase subunit VIII. Mamm. Genome 6, 118-122.
25. MANTHORPE, M., CORNEFERT-JENSEN, F., HARTIKKA, J., FELGNER, J., RUNDELL, A., MARGALITH, M., and DWARKI, V. (1993). Gene therapy by intramuscular injection of plasmid DNA: Studies on firefly luciferase gene expression in mice. Hum. Gene Ther. 4, 419-431.
26. MARTIN, K.A., WALSH, K., and MADER, S.L. (1994). The mouse creatine kinase paired E-box element confers muscle-specific expression to a heterologous promoter. Gene 142, 275-278.
27. MILLER, A.D. (1992a). Human gene therapy comes of age. Nature 357, 455-460.
28. MILLER, A.D. (1992b). Retroviral vectors. Curr. Top. Microbiol. Immunol. 158, 1-24.
29. MILLER, A.D., BENDER, M.A., HARRIS, E.A.S., KALEKO, M., and GELINAS, R.E. (1988). Design of retrovirus vectors for transfer and expression of the human β-globin gene. J. Virol. 62, 4337-4345.
30. OHASHI, T., BOGGS, S., ROBBINS, P., BAHNSON, A., PATRENE, K., WEI, F.S., WEI, J.F., LI, J., LUCHT, L., FEI, Y., CLARK, S., KIMAK, M., HE, H., MOWERY-RUSHTON, P., and BARRANGER, J.A. (1992). Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector. Proc. Natl. Acad. Sci. USA 89, 11332-11336.
31. PALMER, T.D., ROSMAN, G.J., OSBORNE, W.R., and MILLER, A.D. (1991). Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc. Natl. Acad. Sci. USA 88, 1330-1334.
32. PETROPOULOS, C.J., PAYNE, W., SALTER, D.W., and HUGHES, S.H. (1992). Appropriate in vivo expression of a muscle-specific promoter by using avian retroviral vectors for gene transfer. J. Virol. 66, 3391-3397.
33. RAGOT, T., VINCENT, N., CHAFEY, P., VIGNE, E., GILGENKRANTZ, H., COUTON, D., CARTAUD, J., BRIAND, P., KAPLAN, J.C., PERRICARDET, M., and KAHN, A. (1993). Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice. Nature 361, 647-650.
34. RANDO, T.A., and BLAU, H.M. (1994). Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy. J. Cell. Biol. 125, 1275-1287.
35. SALVATORI, G., FERRARI, G., MEZZOGIORNO, A., SERVIDEI, S., COLETTA, M., TONALI, P., GIAVAZZI, R., COSSU, G., and MAVILIO, F. (1993). Retroviral vector-mediated gene transfer into human primary myogenic cells leads to expression in muscle fibers in vivo. Hum. Gene Ther. 4, 713-723.
36. SCHARFMANN, R., AXELROD, J.H., and VERMA, I.M. (1991). Long-term in vivo expression of retrovirus-mediated gene transfer in mouse fibroblast implants. Proc. Natl. Acad. Sci. USA 88, 4626-4630.
37. SCHWARZ, J.J., CHAKRABORTY, T., MARTIN, J., ZHOU, J.M., and OLSON, E.N. (1992). The basic region of myogenin cooperates with two transcription activation domains to induce muscle-specific transcription. Mol. Cell. Biol. 12, 266-275.
38. STERNBERG, E.A., SPIZZ, G., PERRY, W.M., VIZARD, E., WEIL, T., and OLSON, E.N. (1988). Identification of upstream and intragenic regulatory elements that confer cell-type-restricted and differentiation-specific expression on the muscle creatine kinase gene. Mol. Cell. Biol. 8, 2896-2909.
39. TAYLOR, A., ERBA, H.P., MUSCAT, G.E.O., and KEDES, L. (1988). Nucleotide sequence and expression of the human skeletal α-actin gene: Evolution of functional regulatory domains. Genomics 3, 323-336.
40. TRIVEDI, R.A., and DICKSON, G. (1995). Liposome-mediated gene transfer into normal and dystrophin-deficient mouse myoblasts. J. Neurochem. 64, 2230-2238.
41. VINCENT, N., RAGOT, T., GILGENKRANTZ, H., COUTON, D., CHAFEY, P., GREGOIRE, A., BRIAND, P., KAPLAN, J.C., KAHN, A., and PERRICAUDET, M. (1993). Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Nat. Genet. 5, 130-134.
42. VITADELLO, M., SCHIAFFINO, M.V., PICARD, A., SCARPA, M., and SCHIAFFINO, S. (1994). Gene transfer in regenerating muscle. Hum. Gene Ther. 5, 11-18.
43. WEBSTER, C., PAVLATH, G.K., PARKS, D.R., WALSH, F.S., and BLAU, H.M. (1988). Isolation of human myoblasts with the fluorescence-activated cell sorter. Exp. Cell Res. 174, 252-265.
44. WILSON, J.M., JOHNSTON, D.E., JEFFERSON, D.M., and MULLIGAN, R.C. (1988). Correction of the genetic defect in hepatocytes from the Watanabe heritable hyperlipidemic rabbit. Proc. Natl. Acad. Sci. USA 85, 4421-4425.
45. YAO, S.N., and KURACHI, K. (1992). Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc. Natl. Acad. Sci. USA 89, 3357-3361.
46. YAO, S.N., and KURACHI, K. (1993). Implanted myoblasts not only fuse with myofibers but also survive as muscle precursor cells. J. Cell Sci. 105, 957-963.
47. YAO, S.N., WILSON, J.M., NABEL, E.G., KURACHI, S., HACHIYA, H.L., and KURACHI, K. (1991). Expression of human factor IX in rat capillary endothelial cells: Toward somatic gene therapy for hemophilia B. Proc. Natl. Acad. Sci. USA 88, 8101-8105.
48. YAO, S.N., SMITH, K.J., and KURACHI, K. (1994). Primary myoblast-mediated gene transfer persistent expression of human factor IX in mice. Gene Ther. 1, 99-107.