Long-term expression, systemic delivery, and macrophage uptake of recombinant human glucocerebrosidase in mice transplanted with genetically modified primary myoblasts

Human Gene Therapy

Volumn 9, Issue 16, 1998, Pages 2375-2384

  Liu, Chunming ^a   Dunigan, James T ^a   Watkins, Simon C ^b   Bahnson, Alfred B ^a   Barranger, John A ^a,c  

^a GRADUATE SCHOOL OF PUBLIC HEALTH   (United States)

^b UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF PITTSBURGH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; GLUCOSYLCERAMIDASE; RECOMBINANT ENZYME;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BONE MARROW; CELL TRANSPLANTATION; CONTROLLED STUDY; ENZYME BLOOD LEVEL; ENZYME RELEASE; FEMALE; GAUCHER DISEASE; GENE EXPRESSION; GENE TARGETING; GENETIC TRANSDUCTION; IMMUNOHISTOCHEMISTRY; LIVER; MACROPHAGE; MOUSE; MYOBLAST; NONHUMAN; STEM CELL; TARGET ORGAN; TRANSGENE;

EID: 0032212235     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1998.9.16-2375     Document Type: Article

References (11)

1. BAHNSON, A., NIMGAONKAR, M., FEI, Y., BOGGS, S., ROBBINS, P., OHASHI, T., DUNIGAN, J., LI, J., BALL, E.D., and BARRANGER, J.A. (1994). Transduction of CD34+ enriched cord blood and Gaucher bone marrow cells by a retroviral vector carrying the glucocerebrosidase gene. Gene Ther. 1, 176-184.
2. 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. 176, 307-319.
3. BARR, E., and LEIDEN, J.M. (1991). Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254, 1507-1509.
4. BARRANGER, J.A., and GINNS, E.I. (1989). Glucosylceramide lipidoses: Gaucher disease. In The Metabolic Basis of Inherited Disease, Vol. 67. C.R. Scriver, A.L. Beaudet, W.S. Sly, and D. Valle, eds. (McGraw-Hill, New York) pp. 1677-1698.
5. BARRANGER, J.A., OHASHI, T., HONG, C.M., TOMICH, J., AERTS, J.F.G.M., TAGER, J.M., NOLTA, J.A., SENDER, L.S., WEILER, S., and KOHN, D.B. (1989). Molecular pathology and therapy of Gaucher disease. Jpn. J. Inherit. Metab. Dis. 51, 45-71.
6. BARTON, N.W., FURBISH, F.S., MURRAY, G.J., GARFIELD, M., and BRADY, R.O. (1990). Therapeutic response to intravenous infusion of glucocerebrosidase in a patient with Gaucher disease. Proc. Natl. Acad. Sci. U.S.A. 87, 1913-1916.
7. VOGEL, J.C. (1993). Keratinocyte gene therapy. Arch. Dermatol. 129(11), 1478-1483.
8. WATT, D.J. (1990). A comparison of long-term survival of muscle precursor cell suspensions and minced muscle allografts in the non-tolerant mouse. Adv. Exp. Med. Biol. 280, 35-38.
9. WATT, D.J., MORGAN, J.E., and PARTRIDGE, T.A. (1991). Allografts of muscle precursor cells persist in the non-tolerized host. Neuromusc. Disord. 1, 345-355.
10. YAO, S.N., 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(8), 3357-3361.
11. 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(4), 957-963.