A novel endothelial cell-based gene therapy platform for the in vivo delivery of apolipoprotein E

Gene Therapy

Volumn 6, Issue 6, 1999, Pages 1153-1159

  Cioffi, L ^a   Sturtz, F G ^a   Wittmer, S ^a   Barut, B ^a   Smith Gbur, J ^a   Moore, V ^a   Zupancic, T ^a   Gilligan, B ^b   Auerbach, R ^b   Gomez, F ^c   Chauvin, F ^c   Antczak, M ^d   Platika, D ^e   Snodgrass, H R ^a  

^a Progenitor Inc   (United States)

^b UNIVERSITY OF WISCONSIN MADISON   (United States)

^c CENTRE LÉON BÉRARD   (France)

^d UNIVERSITY OF COLORADO   (United States)

^e Ontogeny Inc   (United States)

Author keywords

ApoE; Atherosclerosis; Cardiovascular disease; Endothelial cells; Gene therapy; Yolk sac

Indexed keywords

APOLIPOPROTEIN E; CHOLESTEROL;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; AORTA ATHEROSCLEROSIS; ARTICLE; ATHEROSCLEROSIS; CHOLESTEROL BLOOD LEVEL; CONTROLLED STUDY; EMBRYO; ENDOTHELIUM CELL; GENE TARGETING; GENE THERAPY; MOUSE; NONHUMAN; PRIORITY JOURNAL; SOMATIC CELL GENETICS; VIRUS RECOMBINANT; YOLK SAC;

ANIMALS; APOLIPOPROTEINS E; ARTERIOSCLEROSIS; ENDOTHELIUM; ENZYME-LINKED IMMUNOSORBENT ASSAY; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; MICE; YOLK SAC;

ANIMALIA; MURINAE;

EID: 6544281471     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3300890     Document Type: Article

References (47)

1. Wei Y, Quertermous T, Wagner TE. Directed endothelial differentiation of cultured embryonic yolk sac cells in vivo provides a novel cell-based system for gene therapy. Stem Cells (Dayt) 1995; 13: 541-547.
2. St Louis D, Verma IM. An alternative approach to somatic cell gene therapy. Proc Natl Acad Sci USA 1988; 85: 3150-3154.
3. Palmer TD et al. Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc Natl Acad Sci USA 1991; 88: 1330-1334.
4. Fenjves ES et al. Systemic distribution of apolipoprotein E secreted by grafts of epidermal keratinocyres: implications for epidermal function and gene therapy. Proc Natl Acad Sci USA 1989; 86: 8803-8807.
5. Morgan RA et al. Retroviral vectors containing putative internal ribosome entry sites: development of a polycistronic gene transfer system and applications to human gene therapy. Nucleic Acids Res 1992; 20: 1293-1299.
6. Gerrard AJ et al. Towards gene therapy for haemophilia B using primary human keratinocytes. Nat Genet 1993; 3: 180-183.
7. Smith GH et al. Long-term in vivo expression of genes introduced by retrovirus-mediated transfer into mammary epithelial cells. J Virol 1991; 65: 6365-6370.
8. Zwiebel JA et al. High-level recombinant gene expression in rabbit endothelial cells transduced by retroviral vectors. Science 1989; 243: 220-222.
9. Dai Y et al. Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo. Proc Natl Acad Sci USA 1992; 89: 10892-10895.
10. Lynch CM et al. Long-term expression of human adenosine deaminase in vascular smooth muscle cells of rats: a model for gene therapy. Proc Natl Acad Sci USA 1992; 89: 1138-1142.
11. Berg R. In: Steunberg, Beam, Motulsky, Childs (eds). Progress in Medical Genetics. Saunders: Philadelphia, 1983.
12. Breslow JL. Lipoprotein transport gene abnormalities underlying coronary heart disease susceptibility. Annu Rev Med 1991; 42: 357-371.
13. Grundy SM. George Lyman Duff Memorial Lecture. Multifactorial etiology of hypercholesterolemia. Implications for prevention of coronary heart disease. Arterioscler Thromb 1991; 11: 1619-1635.
14. Lusis AJ. Genetic factors affecting blood lipoproteins: the candidate gene approach. J Lipid Res 1988; 29: 397-429.
15. Getz GS et al. Atherosclerosis and apoprotein E. An enigmatic relationship. Arch Pathol Lab Med 1988; 112: 1048-1055.
16. Mahley RW et al. Plasma lipoproteins: apolipoprotein structure and function. J Lipid Res 1984; 25: 1277-1294.
17. Mahley RW. Apolipoprotein E: cholesterol transport protein with expanding role in cell biology. Science 1988; 240: 622-630.
18. Reue KL et al. Cloning and regulation of messenger RNA for mouse apolipoprotein E. J Biol Chem 1984; 259: 2100-2107.
19. Zannis VI et al. mRNA and apolipoprotein E synthesis abnormalities in peripheral blood monocyte macrophages in familial apolipoprotein E deficiency. J Biol Chem 1985; 260: 12891-12894.
20. Williams DL et al. Apolipoprotein E synthesis in peripheral tissues of nonhuman primates. J Biol Chem 1985; 260: 2444-2451.
21. Driscoll DM, Getz GS. Extrahepatic synthesis of apolipoprotein E. J Lipid Res 1984; 25: 1368-1379.
22. Mahley RW et al. Cellular and molecular biology of lipoprotein metabolism: characterization of lipoprotein receptor-ligand interactions. Cold Spring Harb Symp Quant Biol 1986; 51: 821-828.
23. Nykjaer A et al. The alpha 2-macroglobulin receptor/low density lipoprotein receptor-related protein binds lipoprotein lipase and beta-migrating very low density lipoprotein associated with the lipase. J Biol Chem 1993; 268: 15048-15055.
24. Mulder M et al. Low density lipoprotein receptor internalizes low density and very low density lipoproteins that are bound to heparan sulfate proteoglycans via lipoprotein lipase. J Biol Chem 1993; 268: 9369-9375.
25. Mahley RW et al. Role of heparan sulfate proteoglycans and the LDL receptor-related protein in remnant lipoprotein metabolism. Ann NY Acad Sci 1994; 737: 39-52.
26. Santamarina-Fojo S, Dugi KA. Structure, function and role of lipoprotein lipase in lipoprotein metabolism. Curr Opin Lipidol 1994; 5: 117-125.
27. Takahashi S et al. Rabbit very low density lipoprotein receptor: a low density lipoprotein receptor-like protein with distinct ligand specificity. Proc Natl Acad Sci USA 1992; 89: 9252-9256.
28. Brown MS, Goldstein JL. A receptor-mediated pathway for cholesterol homeostasis. Science 1986; 232: 34-47.
29. Mahley RW, Innerarity TL. Lipoprotein receptors and cholesterol homeostasis. Biochim Biophys Acta 1983; 737: 197-222.
30. Camus MC et al. Distribution and characterization of the serum lipoproteins and apoproteins in the mouse, Mus musculus. J Lipid Res 1983; 24: 1210-1228.
31. Weibust RS. Inheritance of plasma cholesterol levels in mice. Genetics 1973; 73: 303-312.
32. Plump AS et al. Severe hypercholesterolemia and atherosclerosis in apolipoprotein E-deficient mice created by homologous recombination in ES cells. Cell 1992; 71: 343-353.
33. Breslow JL. Transgenic mouse models of lipoprotein metabolism and atherosclerosis. Proc Natl Acad Sci USA 1993; 90: 8314-8318.
34. Linton MF, Atkinson JB, Fazio S. Prevention of atherosclerosis in apolipoprotein E-deficient mice by bone marrow transplantation. Science 1995; 267: 1034-1037.
35. Bellosta S et al. Macrophage-specific expression of human apolipoprotein E reduces atherosclerosis in hypercholesterolemic apolipoprotein E-null mice. J Clin Invest 1995; 96: 2170-2179.
36. Kashyap VS et al. Apolipoprotein E deficiency in mice: gene replacement and prevention of atherosclerosis using adenovirus vectors. J Clin Invest 1995; 96: 1612-1620.
37. Russi TJ, Hirschowitz EA, Crystal RG. Delayed-type hypersensitivity response to high doses of adenoviral vectors. Hum Gene Ther 1997; 8: 323-330.
38. Mahley RW et al. Intravenous infusion of apolipoprotein E accelerates clearance of plasma lipoproteins in rabbits. J Clin Invest 1989; 83: 2125-2130.
39. Shimano H et al. Inhibition of diet-induced atheroma formation in transgenic mice expressing apolipoprotein E in the arterial wall. J Clin Invest 1995; 95: 469-476.
40. Boisvert WA, Spangenberg J, Curtiss LK. Treatment of severe hypercholesterolemia in apolipoprotein E-deficient mice by bone marrow transplantation. J Clin Invest 1995; 96: 1118-1124.
41. Wong GH et al. Interferon-gamma induces enhanced expression of Ia and H-2 antigens on B lymphoid, macrophage, and myeloid cell lines. J Immunol 1983; 131: 788-793.
42. Gretch DG et al. Baculovirus-mediated expression of human apolipoprotein E in Manduca sexta larvae generates particles that bind to the low density lipoprotein receptor. Proc Natl Acad Sci USA 1991; 88: 8530-8533.
43. Felgner PL et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 1987; 84: 7413-7417.
44. Kleinman HK et al. Isolation and characterization of type IV pro-collagen, laminin, and heparan sulfate proteoglycan from the EHS sarcoma. Biochemistry 1982; 21: 6188-6193.
45. Allain CC et al. Enzymatic determination of total serum cholesterol. Clin Chem 1974; 20: 470-475.
46. Cham BE, Knowles BR. A solvent system for delipidation of plasma or serum without protein precipitation. J Lipid Res 1976; 17: 176-181.
47. Paigen B et al. Quantitative assessment of atherosclerotic lesions in mice. Atherosclerosis 1987; 68: 231-240.