Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus–mediated transfer of the VLDL receptor gene (original) (raw)

• Goldstein, J.L., Hobbs, H.H. & Brown, M.S. Familial hypercholesterolemia. in The Metabolic Basis of Inherited Disease(eds Scriver, C.R., Beaudet, A.L., Sly, W.S. & Valle, D.) 1981–2030 (McGraw-Hill, New York, 1995).
  Google Scholar
• Chowdhury, J. et al. Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR deficient rabbits. Science 254, 1802–1805 (1991).
  Article CAS PubMed Google Scholar
• Grossman, M., Raper, S. & Wilson, J. Transplantation of genetically-modified autologus hepatocytes into nonhuman primates: feasibility and short-term toxicity. Hum. Gene Ther. 3, 501–510 (1992).
  Article CAS PubMed Google Scholar
• Grossman, M. et al. Successful Ex Vivo Gene Therapy Directed to Liver Patient with Familial Hypercholesterolemia. Nature Genet. 6, 335–341 (1994).
  Article CAS PubMed Google Scholar
• Grossman, M. et al. A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia. Nature Med. 1148–1154 (1995).
  Article CAS PubMed Google Scholar
• Kozarsky, K. et al. In vivo correction of LDL receptor deficiency in the Watanabe heritable hyperlipidemic rabbit with recombinant adenoviruses. J. Biol. Chem. 269, 13695–13702 (1994).
  CAS PubMed Google Scholar
• Li, J. et al. In vivo gene therapy for hyperlipidemia: phenotypic correction in Watanabe rabbits by hepatic delivery of the rabbit LDL receptor gene. J. Clin. Invest. 95, 768–773 (1995).
  Article CAS PubMed PubMed Central Google Scholar
• Ishibashi, S. et al. Hypercholesterolemia in low density lipoprotein receptor knockout mice and its reversal by adenovirus-mediated gene delivery. J. Clin. Invest. 92, 883–893 (1993).
  Article CAS PubMed PubMed Central Google Scholar
• Yang, Y. et al. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. USA 91, 4407–4411 (1994).
  Article CAS PubMed PubMed Central Google Scholar
• Barr, D. et al. Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains. Gene. Therapy 2, 151–155 (1995).
  CAS PubMed Google Scholar
• Yang, Y., Li, Q., Ertl, H. & Wilson, J. Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J. Virol. 69, 2004–2015 (1995).
  CAS PubMed PubMed Central Google Scholar
• Engelhardt, J., Litzky, L. & Wilson, J. Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a. Hum. Gene Therapy 5, 1217–1229 (1994).
  Article CAS Google Scholar
• Yang, Y., Ertl, H. & Wilson, J. MHC class 1 restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1 deleted recombinant adenoviruses. Immunity 1, 433–442 (1994).
  Article CAS PubMed Google Scholar
• Takahashi, S., Kawarabayasi, Y., Nakai, T., Sakai, J. & Yamamoto, T. Rabbit very low density lipoprotein receptor: a low density lipoprotein receptor-like protein with distinct ligand specificity. Proc. Natl. Acad. Sci. USA 89, 9252–9256 (1992).
  Article CAS PubMed PubMed Central Google Scholar
• Gafvels, M.E. et al. Cloning of a cDNA encoding a putative human very low density lipoprotein/apolipoprotein E receptor and assignment of the gene to chromosome 9pter–p23. Som. Cell Mol. Genet. 19, 557–569 (1993).
  Article CAS Google Scholar
• Gafvels, M.E. et al. Cloning of a complementary deoxyribonucleic acid encoding the murine homolog of the very low density lipoprotein/ apolipoprotein-E receptor: expression pattern and assignment of the gene to mouse chromosome 19. Endocrinol. 135, 387–394 (1994).
  Article CAS Google Scholar
• Oka, K. et al. Mouse very-low-density-lipoprotein receptor (VLDLR) cDNA cloning, tissue-specific expression and evolutionary relationship with the low-density-lipoprotein receptor. J. Biochem. 224, 975, 982 (1994).
  Google Scholar
• Frykman, P., Brown, M., Yamamoto, T., Goldstein, J. & Herz, J. Normal plasma lipoproteins and fertility in gene-targeted mice homozygous for a disruption in the gene encoding very low density lipoprotein receptor. Proc. Natl. Acad. Sci. USA 92, 8453–8457 (1995).
  Article CAS PubMed PubMed Central Google Scholar
• Yamamoto, T., Takahashi, S., Sakai, J. & Kawarabayasi, Y. The very low density lipoprotein receptor, a seond lipoprotein receptor that may mediate uptake of fatty acids into muscle and fat cells. Trends Cardiovasc. Med. 3, 144–148 (1993).
  Article CAS PubMed Google Scholar
• Kass-Eisler, A. et al. The impact of developmental stage, route of administration and the immune system on adenovirus-mediated gene transfer. Gene Tnerapy 1, 395–402 (1994).
  CAS Google Scholar
• Dai, Y. et al. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression. Proc. Natl. Acad. Sci. USA 92, 1401–1405 (1995).
  Article CAS PubMed PubMed Central Google Scholar
• Battey, F. et al. The 39-kDa receptor-associated protein regulates ligand binding by the very low density lipoprotein receptor. J. Biol. Chem. 269, 23268–23273 (1994).
  CAS PubMed Google Scholar
• Van Ginkel, F. et al. Intratracheal gene delivery with adenoviral vector induces elevated systemic IgG and mucosal IgA antibodies to adenovirus and β-galactosidase. Hum. Gene Therapy 6, 895–903 (1995).
  Article CAS Google Scholar
• Yang, Y., Jooss, K.U., Su, Q., Ertl, H.C.J. & Wilson, J.M. Immune responses to viral antigens vs. transgene product in the elimination of recombinant adenovirus infected hepatocytesin vivo. Gene Therapy(in the press).
• Jokinen, E.V. et al. Regulation of the very low density lipoprotein receptor by thyroid hormone in rat skeletal muscle. J. Biol. Chem. 269, 26411–26418 (1994).
  CAS PubMed Google Scholar
• Willnow, T. & Herz, J. Animal models for disorders of hepatic lipoprotein metabolism. J. Mol. Med. 73, 213–20 (1995).
  Article CAS PubMed Google Scholar
• Shimano, H. et al. Secretion-recapture process of apolipoprotein E in hepatic uptake of chylomicron remnants in transgenic mice. J. Clin. Invest. 93, 2215–2223 (1994).
  Article CAS PubMed PubMed Central Google Scholar
• Mokuno, H., Brady, S., Kotite, L., Herz, J. & Havel, R. Effect of the 39-kDa receptor-associated protein on the hepatic uptake and endocytosis of chylomicron remnants and low density lipoproteins in the rat. J. Biol. Chem. 269, 13238–13243 (1994).
  CAS PubMed Google Scholar
• Bu, G., Geuze, H., Strous, G. & Schwartz, A. 39 kDa receptor-associated protein is an ER resident protein and molecular chaperone for LDL receptor-related protein. EMBO J. 14, 2269–2280 (1995).
  Article CAS PubMed PubMed Central Google Scholar
• Polvino, W.J., Dichek, D.A., Mason, J. & Anderson, W.F. Molecular cloning and nucleotide sequence of cDNA encoding a functional murine low-density-lipoprotein receptor. Somat. Cell Mol. Genet. 18, 443–450 (1992).
  Article CAS PubMed Google Scholar
• Hoffer, M.J.V. et al. The mouse low density lipoprotein receptor gene: cDNA sequence and exon–intron structure. Biochem. Biophys. Res. Comm. 191, 880–886 (1993).
  Article CAS PubMed Google Scholar
• Kozarsky, K., Grossman, M. & Wilson, J. Adenovirus-mediated correction of the genetic defect in hepatocytes from patients with familial hypercholesterolemia. Somat. Cell Molec. Genet. 19, 449–458 (1993).
  Article CAS PubMed Google Scholar
• Engelhardt, J. et al. Direct gene transfer of human CFTR into human bronchial epithelia of xenografts with E1 -deleted adenoviruses. Nature Genet. 4, 27–34 (1993).
  Article CAS PubMed Google Scholar
• Yamamoto, T. et al. The human LDL receptor: a cysteine-rich protein with multiple Alu sequences in its mRNA. Cell 39, 27–38 (1984).
  Article CAS PubMed Google Scholar
• Chakrabarti, S., Brechling, K. & Moss, B. Moss, B. vaccinia virus expression vector: coexpression of β-galactosidase provides visual screening of recombinant virus plaques. Mol. Cell. Biol. 5, 3403–3409 (1985).
  Article CAS PubMed PubMed Central Google Scholar
• Wiktor, T. et al. Protection from rabies by a vaccinia virus recombinant containing the rabies virus glycoprotein gene. Proc. Natl. Acad. Sci. USA 81, 7194–7198 (1984).
  Article CAS PubMed PubMed Central Google Scholar