Long-term secretion of therapeutic proteins from genetically modified skeletal muscles

Human Gene Therapy

Volumn 7, Issue 1, 1996, Pages 11-21

  Naffakh, Nadia ^a   Pinset, Christian ^b   Montarras, Didier ^b   Li, Zhenlin ^b   Paulin, Denise ^b   Danos, Olivier ^a   Heard, Jean Michel ^a,b  

^a CNRS   (France)

^b INSTITUT PASTEUR   (France)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLUCURONIDASE; COMPLEMENTARY DNA; DESMIN; ERYTHROPOIETIN; RECOMBINANT PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; ENHANCER REGION; ENZYME ACTIVITY; EXPRESSION VECTOR; GENETIC ENGINEERING; GENETIC TRANSDUCTION; HEMATOCRIT; IMMUNE DEFICIENCY; IMMUNOCOMPETENCE; LIVER DISEASE; LYSOSOME STORAGE DISEASE; MOUSE; NONHUMAN; PROMOTER REGION; PROTEIN EXPRESSION; PROTEIN SECRETION; RETROVIRUS; SKELETAL MUSCLE; SPLEEN DISEASE; TIBIALIS ANTERIOR MUSCLE; TRANSCRIPTION REGULATION;

EID: 0029874105     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1996.7.1-11     Document Type: Article

References (40)

1. ADRA, C.N., BOER, P.H., and McBURNEY, M. (1987). Cloning and expression of the mouse pgk-1 gene and the nucleotide sequence of its promoter. Gene 60, 65-74.
2. BARR, E., and LEIDEN, J.F. (1991). Systemic delivery of recombinant proteins by genetically-modified myoblasts. Science 254, 1507-1509.
3. BAWDEN, C.S., SIVAPRASAD, A.V., VERMA, P.J., WALKER, S.K., and ROGERS, G.E. (1995). Expression of bacterial cysteine biosynthesis genes in transgenic mice and sheep: Toward a new in vivo amino acid biosynthesis pathway and improved wool growth. Transgenic Res. 4, 87-104.
4. BIRKENMEIER, E.H., DAVISSON, M.T., BEAMER, W.G., GANSCHOW, R.E., VOGLER, C.A., GWYNN, B., LYFORD, K.A., MALTAIS, L.M., and WAWRZYNIAK, C.J. (1989). Murine mucopolysaccharidosis type VII. Characterization of a mouse with β-glucuronidase deficiency. J. Clin. Invest. 83, 1258-1266.
5. BIRKENMEIER, E.H., BARKER, J.E., VOGLER, C.A., KYLE, J.W., SLY, W.S., GWYNN, B., LEVY, B., and PEGORS, C. (1991). Increased life span and correction of metabolic defects in murine MPS VII following syngeneic bone marrow transplantation. Blood 78, 3081-3092.
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. Cel. Biol. 7, 887-897.
7. 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.
8. DANOS, O., and MULLIGAN, R.C. (1988). Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc. Natl. Acad. Sci. USA 85, 6460-6464.
9. DAVIS, H.L., WHALEN, R.G., and DEMENEIX, B.A. (1993). Direct gene transfer into skeletal muscle in vivo: Factors affecting efficiency of transfer and stability of expression. Hum. Gene Ther. 4, 151-159.
10. 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-modified myoblasts. Science 254, 1509-1512.
11. DWARKI, V.J., BELLONI, P., NIJJAR, T., SMITH, J., COUTO, L., RABIER, M., CLIFT, S., BERNS, A., and COHEN, L.K. (1994). 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.
12. ENGELHARDT, J.F., KELLUM, M.J., BISAT, F., and PITHA, P.M. (1990). Retrovirus vector-targeted expression of human β-interferon gene to B-cells. Virology 178, 419-428.
13. GLASER, J.H., and SLY, W.S. (1973). β-Glucuronidase deficiency mucopolysaccharidosis: methods for enzymatic diagnosis. J. Lab. Clin. Med. 82, 969-977.
14. HAMAMORI, Y., SAMAL, B., TIAN, J., and KEDES, L. (1994). Persistent erythropoiesis by myoblast transfer of erythropoietin cDNA. Hum. Gene Ther. 5, 1349-1356.
15. KAPLAN, A., ACHORD, D.T., and SLY, W.S. (1977). Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts. Proc. Natl. Acad. Sci. USA 74, 2026-2030.
16. LEROY-VIARD, K., ROUYER-FESSARD, P., and BEUZARD, Y. (1991). Improvement of mouse β-thalassemia by recombinant human erythropoïetin. Blood 78, 1596-1602.
17. LI, Z., and PAULIN, D. (1991). High level desmin expression depends on a muscle-specific enhancer. J. Biol. Chem. 266, 6562-6570.
18. MARÉCHAL, V., NAFFAKH, N., DANOS, O., and HEARD, J.M. (1993). Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically-modified bone marrow cells. Blood 82, 1358-1365.
19. MOULLIER, P., BOHL, D., HEARD, J.M., and DANOS, O. (1993a). Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically-modified skin fibroblasts. Nature Genet. 4, 154-159.
20. MOULLIER, P., MARÉCHAL, V., DANOS, O., and HEARD, J.M. (1993b). Continuous systemic secretion of a lysosomal enzyme by genetically-modified mouse skin fibroblasts. Transplantation 56, 427-432.
21. MOULLIER, P., BOHL, D., CARDOSO, J., HEARD, J.M., and DANOS, O. (1995). Long term delivery of lysosomal enzyme by genetically-modified fibroblasts in dogs. Nature Med. 1, 353-358.
22. NAFFAKH, N., PINSET, C., MONTARRAS, D., PASTORET, C., DANOS, O., and HEARD, J.M. (1994). Transplantation of adult-derived myoblasts in mice following gene transfer. Neuromusc. Disord. 3, 413-417.
23. NAFFAKH, N., VILLEVAL, J.L., MOULLIER, P., HENRI, A., ROUYER-FESSARD, P., DANOS, O., HEARD, J.M., and BEUZARD, Y. (1995). Sustained delivery of erythropoietin in mice by autologous inmplants of genetically-modified skin fibroblasts. Proc. Natl. Acad. Sci. USA 92, 3194-3198.
24. PALMER, T.D., TOMPSON, A.R., and MILLER, A.D. (1989). Production of human factor IX in animals by genetically modified skin fibroblasts: Potential therapy for hemophilia B. Blood 73, 438-445.
25. PFEFFER, S.R. (1991). Targeting of proteins to the lysosome. Curr. Top. Microbiol. Immunol. 170, 43-63.
26. PINSET, C., and MONTARRAS, D. (1994). Cell system for ex vivo studies of myogenesis: a protocol for the isolation of stable muscle cell populations from newborn to adult mice. In Cell Biology: A Laboratory Handbook. J.E. Selis, ed. (Academic Press, Aarhus, Denmark) pp. 199-206.
27. SANDO, G., and NEUFELD, E.F. (1977). Recognition and receptor-mediated uptake of a lysosomal enzyme, α-1-iduronidase, by cultured human fibroblasts. Cell 12, 619-627.
28. SANDS, M.S., BARKER, J.E., VOGLER, C., LEVY, B., GWYNN, B., GALVIN, N., SLY, W.S., and BIRKENMEIER, E. (1993). Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates. Lab. Invest. 68, 676-686.
29. SETOGUCHI, Y., DANEL, C., and CRYSTAL, R.J. (1994). Stimulation of erythropoiesis by in vivo gene therapy: physiologic consequences of transfer of the human eryhtropoietin gene using an adenovirus vector. Blood 84, 2946-2953.
30. SLY, W.S. (1980). Multiple recognition forms of human β-glucuronidase and their pinocytosis receptors: Implications for enzyme therapy. Birth Defects 16, 115-128.
31. SORIANO, P., FRIEDRICH, G., and LAWINGER, P. (1991). Promoter interactions in retrovirus vectors introduced into fibroblasts and embryonic stem cells. J. Virol. 65, 2314-2319.
32. TRIPATHY, S.K., GOLDWASSER, E., LU, M.M., BARR, E., and LEIDEN, J.M. (1994). Stable delivery of physiologic levels of recombinant erythropoietin to the systemic circulation by intramuscular injection of replication-defective adenovirus. Proc. Natl. Acad. Sci. USA 91, 11557-11561.
33. ULLRICH, R., MERSMANN, G., WEBER, E., and VON FIGURA, K. (1978). Evidence for lysosomal recognition by human fibroblasts via a phosphorylated carbohydrate moiety. Biochem. J. 170, 643-650
34. VILE, R., MILLER, N., CHERNAJOVSKY, Y., and HART, I. (1994). A comparison of the properties of different retroviral vectors containing the murine tyrosinase promoter to achieve transcriptionally targeted expression of the HSVtk or IL2 genes. Gene Ther. 1, 307-316.
35. VOGLER, C., BIRKENMEIER, E.H., SLY, W.S., LEVY, B., PEGORS, C., KYLE, J.W., and BEAMER, W.G. (1990). A murine model of mucopolysaccharidosis VII: Gross and microscopic findings in beta-glucuronidase-deficient mice. Am. J. Pathol. 136, 207-217.
36. VOGLER, C., SANDS, M., HIGGINS, A., LEVY, B., GRUBB, J., BIRKENMEIER, E.H., and SLY, W.S. (1993). Enzyme replacement with recombinant β-glucuronidase in the newborn mucopolysaccharidosis type VII mouse. Pediatr. Res. 34, 837-840.
37. WOLFE, J.H., SANDS, M.S., BARKER, J.E., GWYNN, B., ROWE, L.B., VOGLER, C.A., and BIRKENMEIER, E.H. (1992). Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature 360, 749-753.
38. 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.
39. YEE, J.K., MOORES, J., JOLLY, D., WOLLF, J., RESPESS, J., and FRIEDMANN, T. (1987). Gene expression from transcriptionally disabled retroviral vectors. Proc. Natl. Acad. Sci. USA 84, 5199-5201.
40. YU, S., VON RUDEN, T., KANTOFF, P.W., GARBER, C., SEIBERG, M., RÜTHER, U., ANDERSON, W.F., WAGNER, E.F., and GILBOA, E. (1986). Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc. Natl. Acad. Sci. 83, 3194-3198.