Tissue-engineered human bioartificial muscles expressing a foreign recombinant protein for gene therapy

Human Gene Therapy

Volumn 10, Issue 4, 1999, Pages 565-577

  Powell, Courtney ^a   Shansky, Janet ^b   Del Tatto, Michael ^b   Forman, Daniel E ^c   Hennessey, James ^d   Sullivan, Kathryn ^b   Zielinski, Beth A ^b   Vandenburgh, Herman H ^a,b,e  

^a BROWN UNIVERSITY   (United States)

^b BROWN UNIVERSITY   (United States)

^c MIRIAM HOSPITAL   (United States)

^d RHODE ISLAND HOSPITAL   (United States)

^e Cell Based Delivery Inc   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GROWTH FACTOR; RECOMBINANT GROWTH HORMONE; RECOMBINANT PROTEIN; VIRUS VECTOR;

ADULT; AGED; ARTICLE; CELL DIFFERENTIATION; CELL FUSION; CELL ISOLATION; CONGESTIVE HEART FAILURE; EXPRESSION VECTOR; FEMALE; GENE THERAPY; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; KINETICS; MALE; MOLECULAR CLONING; MUSCLE BIOPSY; MUSCLE CELL; MYOBLAST; RETROVIRUS; SKELETAL MUSCLE; STEM CELL;

NASA DISCIPLINE MUSCULOSKELETAL; NON-NASA CENTER;

ADULT; AGED; AGED, 80 AND OVER; ARTIFICIAL ORGANS; CELL DIFFERENTIATION; CELL DIVISION; FEMALE; GENE THERAPY; GROWTH HORMONE; HUMANS; IMMUNOHISTOCHEMISTRY; MALE; MIDDLE AGED; MUSCLE, SKELETAL; RADIOIMMUNOASSAY; RECOMBINANT PROTEINS; TRANSDUCTION, GENETIC;

MURINAE; UNIDENTIFIED RETROVIRUS;

EID: 0033104799     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950018643     Document Type: Article

References (52)

1. ASKANAS, V., ENGEL, W.K., BRITTON, D.E., ADORNATO, B.T., and EIBEN, R.M. (1978). Reincarnation in cultured muscle of mitochondrial abnormalities from two patients with epilepsy and lactic acidosis. Arch. Neurol. 35, 801-809.
2. BARR, E., and LEIDEN, J.M. (1991). Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254, 1507-1509.
3. BAUMANN, G. (1991). Hormone heterogeneity: Genes, isohormones, variants, and binding proteins. Endocr. Rev. 12, 424-449.
4. BEHARA, A.M., WESTCOTT, A.J., and CHANG, P.L. (1992). Intrathymic implants of genetically modified fibroblasts. FASEB J. 6, 2853-2858.
5. BELLES-ISLES, M., ROY, R., DANSEREAU, G., GOULET, M., ROY, B., BOUCHARD, J.P., and TREMBLAY, J.P. (1993). Rapid selection of donor myoblast clones for muscular dystrophy therapy using cell surface expression of NCAM. Eur. J. Histochem. 37, 375-380.
6. BLAU, H.M., and SPRINGER, M.L. (1995a). Gene therapy - a novel form of drug delivery. N. Engl. J. Med. 333, 1204-1207.
7. BLAU, H.M., and SPRINGER, M.L. (1995b). Muscle-mediated gene therapy. N. Engl. J. Med. 333, 1554-1556.
8. BLAU, H.M., and WEBSTER, C. (1981). Isolation and characterization of human muscle cells. Proc. Natl. Acad. Sci. U.S.A. 78, 5623-5627.
9. BOHL, D., NAFFAKH, N., and HEARD, J.M. (1997). Long-term control of erythropoietin secretion by doxycycline in mice transplanted with engineered primary myoblasts. Nature Med. 3, 299-305.
10. BONHAM, L., PALMER, T., and MILLER, A.D. (1996). Prolonged expression of therapeutic levels of human granulocyte colony-stimulating factor in rats following gene transfer to skeletal muscle. Hum. Gene Ther. 7, 1423-1429.
11. CHEN, B.-F., CHANG, W.-C., CHEN, S.-T., CHEN, D.-S., and HWANG, L.-H. (1995). Long-term expression of the biologically active growth hormone in genetically modified fibroblasts after implantation into a hypophysectomized rat. Hum. Gene Ther. 6, 917-926.
12. CHROMIAK, J., SHANSKY, J., PERRONE, C.E., and VANDENBURGH, H.H. (1998). Bioreactor perfusion system for the long term maintenance of tissue-engineered skeletal muscle organoids. In Vitro Cell Dev. Biol. 34, 694-703.
13. DANOS, O., and MULLIGAN, R.C. (1988). Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc. Natl. Acad. Sci. U.S.A. 85, 6460-6464.
14. DECARY, S., MOULY, V., BEN HAMIDA, C., SAUTET, A., BARBET, J.P., and BUTLER-BROWNE, G.S. (1997). Replicative potential and telomere length in human skeletal muscle: Implications for satellite cell-mediated gene therapy. Hum. Gene Ther. 8, 1429-1438.
15. 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.
16. DUERR, R.L., HUANG, S., MIRALIAKBAR, H.R., CLARK, R., CHIEN, K.R., and ROSS, J., JR. (1995). Insulin-like growth factor-1 enhances ventricular hypertrophy and function during the onset of experimental cardiac failure. J. Clin. Invest. 95, 619-627.
17. FAZIO, S., SABATINI, D., CAPALDO, B., VIGORITO, C., GIORDANO, A., GUIDA, R., PARDO, F., BIONDI, B., and SACCÀ, L. (1996). A preliminary study of growth hormone in the treatment of dilated cardiomyopathy. N. Engl. J. Med. 334, 809-814.
18. GUENARD, V., KLEITMAN, N., MORRISSEY, T.K., BUNGE, R.P., and AEBISCHER, P. (1992). Syngeneic Schwann cells derived from adult nerves seeded in semipermeable guidance channels enhance peripheral nerve regeneration. J. Neurosci. 12, 3310-3320.
19. HAM, R.G., ST. CLAIR, J.A., WEBSTER, C., and BLAU, H.M. (1988). Improved media for normal human muscle satellite cells: Serum-free clonal growth and enhanced growth with low serum. In Vitro Cell. Dev. Biol. 24, 833-844.
20. HAMAMORI, Y., SAMAL, B., TIAN, J., and KEDES, L. (1994). Persistent erythropoiesis by myoblast transfer of erythropoietin cDNA. Hum. Gene Ther. 5, 1349-1356.
21. 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.
22. HARVEY, S., and DAUGHADAY, W.H. (1995). Growth hormone release: Profiles. In Growth Hormone, 1st Ed. S. Harvey, C.G. Scanes, and W.H. Daughaday, eds. (CRC Press, Boca Raton, FL) pp. 193-223.
23. HAUSCHKA, S.D., LINKHARD, T.A., CLEGG, C., and MERRILL, G. (1979). Clonal studies of human and mouse muscle. In Muscle Regeneration. A. Mauro, ed. (Raven Press, New York) pp. 311-322.
24. HENNESSEY, J.V., CHROMIAK, J.A., DELLA VENTURA, S., GUERTIN, J., and MACLEAN, D.B. (1997). Increase in percutaneous muscle biopsy yield with a suction-enhancement technique. J. Appl. Physiol. 82, 1739-1742.
25. JIAO, S., WILLIAMS, P., BERG, R.K., HODGEMAN, B.A., LIU, L., REPETTO, G., and WOLFF, J.A. (1992). Direct gene transfer into nonhuman primate myofibers in vivo. Hum. Gene Ther. 3, 21-33.
26. LANGER, R., and VACANTI, J.P. (1993). Tissue engineering. Science 260, 920-926.
27. LOMBARDI, G., COLAO, A., FERONE, D., MARZULLO, P., ORIO, F., LONGOBARDI, S., and MEROLA, B. (1997). Effect of growth hormone on cardiac function. Horm. Res. 48, 38-42.
28. MANCINI, D.M., WALTER, G., REICHNEK, N., LENKINSKI, R., MCCULLY, K.K., MULLEN, J.L., and WILSON, J.R. (1992). Contribution of skeletal muscle atrophy to exercise intolerance and altered muscle metabolism in heart failure. Circulation 85, 1364-1673.
29. MASSIE, B.M., SIMONINI, A., SAHGAL, P., WELLS, L., and DUDLEY, G.A. (1996). Relation of systemic and local muscle exercise capacity to skeletal muscle characteristics in men with congestive heart failure. J. Am. Coll. Cardiol. 27, 140-145.
30. MILLER, A.D., MILLER, D.G., GARCIA, J.V., and LYNCH, C.M. (1993). Use of retroviral vectors for gene transfer and expression. Methods Enzymol. 217, 581-599.
31. MOONEY, D.J., and ROWLEY, J.A. (1997). Tissue engineering: Integrating cells and materials to create functional tissue replacements. In Controlled Drug Delivery: Challenges and Strategies. Kinam Park, ed. (ACS, Washington, D.C.) pp. 333-346.
32. MORGAN, J.R., BARRANDON, Y., GREEN, H., and MULLIGAN, R.C. (1986). Expression of an exogenous growth hormone gene by transplantable human epidermal cells. Science 237, 1476-1478.
33. NEUMEYER, A.M., DIGREGORIO, D.M., and BROWN, R.H., JR. (1992). Arterial delivery of myoblasts to skeletal muscle. Neurology 42, 2258-2262.
34. SANDERSON, R.D., FITCH, J.M., LINSENMAYER, T.R., and MAYNE, R. (1986). Fibroblasts promote the formation of a continuous basal lamina during myogenesis in vitro. J. Cell Biol. 102, 740-747.
35. SHANSKY, J., DEL TATTO, M., CHROMIAK, J., and VANDENBURGH, H. (1997). A simplified method for tissue engineering skeletal muscle organoids in vitro. In Vitro Cell. Dev. Biol. Anim. 33, 659-661.
36. SIPEHIA, R., GARFINKLE, A., JACKSON, W.B., and CHANG, T.M. (1990). Towards an artificial cornea: Surface modifications of optically clear, oxygen permeable soft contact lens materials by ammonia plasma modification technique for the enhanced attachment and growth of corneal epithelial cells. Biomaterials Artificial Cells Artificial Organs 18, 643-655.
37. SPRINGER, M.L., and BLAU, H.M. (1997). High-efficiency retroviral infection of primary myoblasts. Somat. Cell Mol. Genet. 23, 203-209.
38. 2+ handling and enhance cardiac performance. Circ. Res. 79, 227-236.
39. SULLIVAN, M.J., GREEN, H.J., and COBB, F.R. (1990). Skeletal muscle biochemistry and histology in ambulatory patients with long-term heart failure. Circulation 81, 518-527.
40. TAYLOR, S.H. (1996). Congestive heart failure. Eur. Heart J. 17B, 43-56.
41. THOMPSON, K.P., HANNA, K., WARING, G.O., GIPSON, I., LIU, Y., GAILITIS, R.P., JOHNSON-WINT, B., and GREEN, K. (1991). Current status of synthetic epikeratoplasty. Refractive Corneal Surg. 7, 240-248.
42. TRINKAUS-RANDALL, V., CAPECCHI, J., NEWTON, A., VADASZ, A., LEIBOWITZ, H., and FRANZBLAU, C. (1988). Development of a biopolymeric keratoprosthetic material. Evaluation in vitro and in vivo. Invest. Opthalmol. Vis. Sci. 29, 393-400.
43. VANDENBURGH, H.H., SWASDISON, S., and KARLISCH, P. (1991). Computer aided mechanogenesis of skeletal muscle organs from single cells in vitro. FASEB J. 5, 2860-2867.
44. VANDENBURGH, H.H., SOLERSSI, R., SHANSKY, J., ADAMS, J.W., and HENDERSON, S.A. (1996a). Mechanical stimulation of organogenic cardiomyocyte growth in vitro. Am. J. Physiol. Cell Physiol. 270, C1284-C1292.
45. VANDENBURGH, H.H., DEL TATTO, M., SHANSKY, J., LEMAIRE, J., CHANG, A., PAYUMO, F., LEE, P., GOODYEAR, A., and RAVEN, L. (1996b). Tissue engineered skeletal muscle organoids for reversible gene therapy. Hum. Gene Ther. 7, 2195-2200.
46. VANDENBURGH, H.H., DEL TATTO, M., SHANSKY, J., GOLDSTEIN, L., and RUSSELL, K., GENES, N., CHROMIAK, J., and YAMADA, S. (1998). Attenuation of skeletal muscle wasting with recombinant human growth hormone secreted from a tissue engineered bioartificial muscle. Hum. Gene Ther. 9, 2555-2564.
47. VAN DER VENN, P.F.M., SCHAART, G., JAP, P.H.K., SENGERS, R.C.A., STADHOUDERS, A.M., and RAMAEKERS, C.S. (1992). Differentiation of human skeletal muscle cells in culture: Maturation as indicated by titin and desmin striation. Cell Tissue Res. 270, 189-198.
48. WEBSTER, C., and BLAU, H.M. (1990). Accelerated age-related decline in replicative life-span of Duchenne muscular dystrophy myoblasts: Implications for cell and gene therapy. Somatic Cell Mol. Genet. 16, 557-565.
49. 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.
50. YANG, R., BUNTING, S., GILLETT, N., CLARK, R., and JIN, H. (1995). Growth hormone improves cardiac performance in experimental heart failure. Circulation 92, 262-267.
51. YAO, S., and KURACHI, K. (1992). Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc. Natl. Acad. Sci. U.S.A. 89, 3357-3361.
52. YOO, J.J., and ATALA, A. (1997). A novel gene delivery system using urothelial tissue engineered neo-organs. J. Urol. 158, 1066-1070.