Long-term expression and secretion of human glucocerebrosidase by primary murine and human myoblasts and differentiated myotubes

Journal of Molecular Medicine

Volumn 76, Issue 11, 1998, Pages 773-781

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

^a UNIVERSITY OF PITTSBURGH GRADUATE SCHOOL OF PUBLIC HEALTH   (United States)

^b UNIVERSITY OF PITTSBURGH   (United States)

^c HUAZHONG UNIVERSITY OF SCIENCE AND TECHNOLOGY   (China)

Author keywords

Bone marrow derived macrophage; Gaucher disease; Glucocerebrosidase; Myoblast; Retroviral vector

Indexed keywords

DNA; GLUCOSYLCERAMIDASE;

ANIMAL CELL; CONTROLLED STUDY; ENZYME ACTIVITY; ENZYME RELEASE; EXPRESSION VECTOR; FEMALE; GAUCHER DISEASE; GENE THERAPY; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; IMMUNOCYTOCHEMISTRY; MACROPHAGE; MOUSE; MYOBLAST; MYOTUBE; NONHUMAN; POLYMERASE CHAIN REACTION; RETROVIRUS; REVIEW;

ANIMALS; BONE MARROW CELLS; CELL DIFFERENTIATION; CELL TRANSFORMATION, VIRAL; CELLS, CULTURED; COCULTURE TECHNIQUES; EXTRACELLULAR SPACE; FEMALE; GENE EXPRESSION; GLUCOSYLCERAMIDASE; HUMANS; INTRACELLULAR FLUID; MACROPHAGES; MICE; MICE, INBRED C3H; MUSCLE, SKELETAL; TIME FACTORS;

EID: 0031670323     PISSN: 09462716     EISSN: None     Source Type: Journal    
DOI: 10.1007/s001090050279     Document Type: Review

References (26)

1. Patrick AD (1965) A deficiency of glucocerebrosidase in Gaucher's disease. Biochem J 97:17C
2. Brady RO, Kanfer JN, Shapiro D (1965) Metabolism of glucocerebrosides. II. Evidence of an enzymatic deficiency in Gaucher disease. Biochem Biophys Res Commun 18:221-225
3. Barranger JA, Ginns EI (1989) Glucosylceramide lipidoses: Gaucher disease. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The metabolic basis of inherited disease, vol 67. McGraw-Hill, New York, pp 1677-1698
4. Barranger JA, Ohashi T, Hong CM, Tomich J, Aerts JFGM, Tager JM, Nolta JA, Sender LS, Weiler S, Kohn DB (1989) Molecular pathology and therapy of Gaucher disease. Jpn J Inherit Metab Dis 51:45-71
5. Furbish FS, Steer CJ, Barranger JA, Jones EA, Brady RO (1978) The uptake of native and desialylated glucocerebrosidase by rat hepatocytes and Kupffer cells. Biochem Biophys Res Commun 81:1047-1053
6. Furbish FS, Steer CJ, Krett NL, Barranger JA (1981) Uptake and distribution of placental glucocerehrosidase in rat hepatic cells and effects of sequential deglycosylation. Biochim Biophys Acta 673:425-434
7. Barton NW, Furbish FS, Murray GJ, Garfield M, Brady RO (1990) Therapeutic response to intravenous infusion of glucocerebrosidase in a patient with Gaucher disease. Proc Natl Acad Sci USA 87:1913-1916
8. Barton NW, Brady RO, Dambrosia JM (1992) Dose-dependent response to marcophage-targeted gluocerebrosidase for Gaucher disease. J Pediatr 20:277-280
9. Frickson AH, Ginns FI, Barranger JA (1985) Biosynthesis of the lysosomal enzyme glucocerebrosidase. J Biol Chem 260: 14319-14324
10. van Dongen JM, Willemsen R, Ginns EI, Sips HJ, Tager JM, Barranger JA, Reuser AJ (1986) The subcellular localization of soluble and membrane-bound lysosomal enzymes in I-cell fibroblasts: A comparative immunocytochemical study. Eur J Cell Biol 39:179-186
11. Bansal V, Mowery-Rushton P, Lucht L, Li J, Bahnson A, Watkins SC, Barranger JA (1994) Transduction, expression, and secretion of human glucocerehrosidase by murine myoblasts. Ann NY Acad Sci 176:307-319
12. Barr E, Leiden JM (1991) Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254:1507-1509
13. Dhawan J, Pan LC, Pavlath GK, Travis MA, Lanctot AM, Blau HM (1991) Systemic delivery of human growth hormone by injection of genetically engineering myoblasts. Science 254:1509-1512
14. Rando TA, Blau HM (1994) Primary mouse myoblast purification, charcterization, and transplantation for cell-mediated gene therapy. J Cell Biol 125:1275-1287
15. Naffakh N, Pinset C, Montarras D, Pastoret C, Danos O, Heard JM (1993) Transplantation of adult-derived myoblasts in mice following gene transfer. Neuromuscul Disord 3:413-417
16. Dai Y, Roman M, Naviaux RK, Verma IM (1992) Gene therapy via primary myoblasts: long-term expression of factors IX protein following transplantation in vivo. Proc Natl Acad Sci USA 89:10892-10895
17. Roman M, Axelrod JH, Dai Y, Naviaux RK, Friedmann T, Verma IM (1992) Circulating human or canine factor IX from retrovirally transduced primary myoblasts and established myoblast cell lines grafted into murine skeletal muscle. Somat Cell Mol Genet 18:247-258
18. Yao SH, Kurachi K (1992) Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc Natl Acad Sci USA 89:3357-3361
19. Ohashi T, Boggs S, Robbins P, Bahnson A, Patrene K, Wei FS, Wei JF, Lucht L, Fei Y, Clark S, Kimak M, He H, Mowery-Rushton P, Barranger JA. (1992) Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and the functional macrophages following transplantation of bone marrow transduced by retroviral vector. Proc Natl Acad Sci USA 89:11332-11336
20. Danos O, Mulligan RC (1988) Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc Natl Acad Sci USA 85:6460-6464
21. Gregory SH (1988) Substratum-dependent proliferation and survival of bone marrow-derived mononuclear phagocytes. J Leukoc Biol 43:57-79
22. Cepko C (1996) Transduction of genes using retrovirus vectors. In: Current protocols in molecular biology, supplement 36:9.10.9-10. Wiley, New York
23. Kim HS, Smithies O (1988) recombinant fragment assay for gene targeting based on the polymerase chain reaction. Nucleic Acid Res 16:8887-8903
24. Murray GJ (1987) Lectin-specific targeting of lysosomal enzyme to reticuloendothelial cells. Methods Enzymol 149:25-42
25. Murray GJ, Jin F-S (1995) Immunoelectron-icroscopic localization of mannose-terminal glucocerebrosidase in lysosomes of rat liver Kuppfer cells. J Histochem Cytochem 43:149-158
26. Liu C, Dunigan J, Watkins SC, Bahnson AB, Barranger JB (1998) Long-term expression, systemic delivery and macrophage uptake of recombinant human glucocerebrosidase in mice transplanted with genetical modified primary myoblasts. Hum Gene Ther (in press)