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Volumn 35, Issue 1, 2016, Pages 50-59

Induction of sustained hypercholesterolemia by single adeno-associated virus-mediated gene transfer of mutant hPCSK9

Author keywords

Atherosclerosis; hypercholesterolemia; PCSK9

Indexed keywords

APOLIPOPROTEIN E; LOW DENSITY LIPOPROTEIN; BIOLOGICAL MARKER; CHOLESTEROL; PCSK9 PROTEIN, HUMAN; SERINE PROTEINASE;

EID: 84920140051     PISSN: 10795642     EISSN: 15244636     Source Type: Journal    
DOI: 10.1161/ATVBAHA.114.303617     Document Type: Article
Times cited : (136)

References (49)
  • 1
    • 82255192241 scopus 로고    scopus 로고
    • Can we dramatically reduce the incidence of coronary heart disease?
    • Domanski M, Lloyd-Jones D, Fuster V, Grundy S. Can we dramatically reduce the incidence of coronary heart disease? Nat Rev Cardiol. 2011;8:721-725.
    • (2011) Nat Rev Cardiol. , vol.8 , pp. 721-725
    • Domanski, M.1    Lloyd-Jones, D.2    Fuster, V.3    Grundy, S.4
  • 3
    • 79955535643 scopus 로고    scopus 로고
    • Macrophages in the pathogenesis of atherosclerosis
    • Moore KJ, Tabas I. Macrophages in the pathogenesis of atherosclerosis. Cell. 2011;145:341-355.
    • (2011) Cell. , vol.145 , pp. 341-355
    • Moore, K.J.1    Tabas, I.2
  • 4
    • 0035936802 scopus 로고    scopus 로고
    • Atherosclerosis. The road ahead
    • Glass CK, Witztum JL. Atherosclerosis. the road ahead. Cell. 2001;104:503-516.
    • (2001) Cell. , vol.104 , pp. 503-516
    • Glass, C.K.1    Witztum, J.L.2
  • 5
    • 0028268018 scopus 로고
    • The two-receptor model of lipoprotein clearance: Tests of the hypothesis in "knockout" mice lacking the low density lipoprotein receptor, apolipoprotein E, or both proteins
    • Ishibashi S, Herz J, Maeda N, Goldstein JL, Brown MS. The two-receptor model of lipoprotein clearance: tests of the hypothesis in "knockout" mice lacking the low density lipoprotein receptor, apolipoprotein E, or both proteins. Proc Natl Acad Sci USA. 1994;91:4431-4435.
    • (1994) Proc Natl Acad Sci USA. , vol.91 , pp. 4431-4435
    • Ishibashi, S.1    Herz, J.2    Maeda, N.3    Goldstein, J.L.4    Brown, M.S.5
  • 6
    • 0026725757 scopus 로고
    • Severe hypercholesterolemia and atherosclerosis in apolipoprotein E-defcient mice created by homologous recombination in ES cells
    • Plump AS, Smith JD, Hayek T, Aalto-Setälä K, Walsh A, Verstuyft JG, Rubin EM, Breslow JL. Severe hypercholesterolemia and atherosclerosis in apolipoprotein E-defcient mice created by homologous recombination in ES cells. Cell. 1992;71:343-353.
    • (1992) Cell. , vol.71 , pp. 343-353
    • Plump, A.S.1    Smith, J.D.2    Hayek, T.3    Aalto-Setälä, K.4    Walsh, A.5    Verstuyft, J.G.6    Rubin, E.M.7    Breslow, J.L.8
  • 7
    • 0027258140 scopus 로고
    • Hypercholesterolemia in low density lipoprotein receptor knockout mice and its reversal by adenovirus-mediated gene delivery
    • Ishibashi S, Brown MS, Goldstein JL, Gerard RD, Hammer RE, Herz J. Hypercholesterolemia in low density lipoprotein receptor knockout mice and its reversal by adenovirus-mediated gene delivery. J Clin Invest. 1993;92:883-893.
    • (1993) J Clin Invest. , vol.92 , pp. 883-893
    • Ishibashi, S.1    Brown, M.S.2    Goldstein, J.L.3    Gerard, R.D.4    Hammer, R.E.5    Herz, J.6
  • 10
    • 2442670243 scopus 로고    scopus 로고
    • Mutations in the PCSK9 gene in Norwegian subjects with auto-somal dominant hypercholesterolemia
    • Leren TP. Mutations in the PCSK9 gene in Norwegian subjects with auto-somal dominant hypercholesterolemia. Clin Genet. 2004;65:419-422.
    • (2004) Clin Genet. , vol.65 , pp. 419-422
    • Leren, T.P.1
  • 11
    • 84879525224 scopus 로고    scopus 로고
    • Is the future of statins aligned with new novel lipid modulation therapies?
    • Phan BA, Toth PP. Is the future of statins aligned with new novel lipid modulation therapies? Curr Atheroscler Rep. 2013;15:300.
    • (2013) Curr Atheroscler Rep. , vol.15 , pp. 300
    • Phan, B.A.1    Toth, P.P.2
  • 13
    • 2342451128 scopus 로고    scopus 로고
    • Adenoviral-mediated expression of Pcsk9 in mice results in a low-density lipoprotein receptor knockout phenotype
    • Maxwell KN, Breslow JL. Adenoviral-mediated expression of Pcsk9 in mice results in a low-density lipoprotein receptor knockout phenotype. Proc Natl Acad Sci USA. 2004;101:7100-7105.
    • (2004) Proc Natl Acad Sci USA. , vol.101 , pp. 7100-7105
    • Maxwell, K.N.1    Breslow, J.L.2
  • 15
    • 84864770647 scopus 로고    scopus 로고
    • Identifcation and characterization of new gain-of-function mutations in the PCSK9 gene responsible for auto-somal dominant hypercholesterolemia
    • Abifadel M, Guerin M, Benjannet S, et al. Identifcation and characterization of new gain-of-function mutations in the PCSK9 gene responsible for auto-somal dominant hypercholesterolemia. Atherosclerosis. 2012;223:394-400.
    • (2012) Atherosclerosis. , vol.223 , pp. 394-400
    • Abifadel, M.1    Guerin, M.2    Benjannet, S.3
  • 16
    • 84866499818 scopus 로고    scopus 로고
    • Pcsk9 sirna suppresses the infammatory response induced by oxldl through inhibition of nf-kappab activation in thp-1-derived macrophages
    • Tang Z, Jiang L, Peng J, Ren Z, Wei D, Wu C, Pan L, Jiang Z, Liu L. Pcsk9 sirna suppresses the infammatory response induced by oxldl through inhibition of nf-kappab activation in thp-1-derived macrophages. Int J Mol Med. 2012;30:931-938
    • (2012) Int J Mol Med. , vol.30 , pp. 931-938
    • Tang, Z.1    Jiang, L.2    Peng, J.3    Ren, Z.4    Wei, D.5    Wu, C.6    Pan, L.7    Jiang, Z.8    Liu, L.9
  • 18
    • 34247605063 scopus 로고    scopus 로고
    • Structural and biophysical studies of PCSK9 and its mutants linked to familial hypercholesterol-emia
    • Cunningham D, Danley DE, Geoghegan KF, et al. Structural and biophysical studies of PCSK9 and its mutants linked to familial hypercholesterol-emia. Nat Struct Mol Biol. 2007;14:413-419.
    • (2007) Nat Struct Mol Biol. , vol.14 , pp. 413-419
    • Cunningham, D.1    Danley, D.E.2    Geoghegan, K.F.3
  • 19
    • 84857602982 scopus 로고    scopus 로고
    • Adeno-associated virus vectorology, manufacturing, and clinical applications
    • Grieger JC, Samulski RJ. Adeno-associated virus vectorology, manufacturing, and clinical applications. Methods Enzymol. 2012;507:229-254.
    • (2012) Methods Enzymol. , vol.507 , pp. 229-254
    • Grieger, J.C.1    Samulski, R.J.2
  • 20
    • 84892924162 scopus 로고    scopus 로고
    • Long-term effects of AAV1/SERCA2a gene transfer in patients with severe heart failure: Analysis of recurrent cardiovascular events and mortality
    • Zsebo K, Yaroshinsky A, Rudy JJ, Wagner K, Greenberg B, Jessup M, Hajjar RJ. Long-term effects of AAV1/SERCA2a gene transfer in patients with severe heart failure: analysis of recurrent cardiovascular events and mortality. Circ Res. 2014;114:101-108.
    • (2014) Circ Res. , vol.114 , pp. 101-108
    • Zsebo, K.1    Yaroshinsky, A.2    Rudy, J.J.3    Wagner, K.4    Greenberg, B.5    Jessup, M.6    Hajjar, R.J.7
  • 21
    • 84897570851 scopus 로고    scopus 로고
    • Recombinant AAV as a platform for translating the therapeutic potential of RNA interference
    • Borel F, Kay MA, Mueller C. Recombinant AAV as a platform for translating the therapeutic potential of RNA interference. Mol Ther. 2014;22:692-701.
    • (2014) Mol Ther. , vol.22 , pp. 692-701
    • Borel, F.1    Kay, M.A.2    Mueller, C.3
  • 23
    • 84887018396 scopus 로고    scopus 로고
    • Proprotein convertases subtilisin/kexin type 9, an enzyme turned escort protein: Hepatic and extra hepatic functions
    • Mbikay M, Mayne J, Chrétien M. Proprotein convertases subtilisin/kexin type 9, an enzyme turned escort protein: hepatic and extra hepatic functions. J Diabetes. 2013;5:391-405.
    • (2013) J Diabetes. , vol.5 , pp. 391-405
    • Mbikay, M.1    Mayne, J.2    Chrétien, M.3
  • 25
    • 60749122013 scopus 로고    scopus 로고
    • Degradation of LDLR protein mediated by 'gain of function' PCSK9 mutants in normal and ARH cells
    • Fasano T, Sun XM, Patel DD, Soutar AK. Degradation of LDLR protein mediated by 'gain of function' PCSK9 mutants in normal and ARH cells. Atherosclerosis. 2009;203:166-171.
    • (2009) Atherosclerosis. , vol.203 , pp. 166-171
    • Fasano, T.1    Sun, X.M.2    Patel, D.D.3    Soutar, A.K.4
  • 27
    • 0037603589 scopus 로고    scopus 로고
    • Mutations in PCSK9 cause autoso-mal dominant hypercholesterolemia
    • Abifadel M, Varret M, Rabès JP, et al. Mutations in PCSK9 cause autoso-mal dominant hypercholesterolemia. Nat Genet. 2003;34:154-156.
    • (2003) Nat Genet. , vol.34 , pp. 154-156
    • Abifadel, M.1    Varret, M.2    Rabès, J.P.3
  • 28
    • 84872013355 scopus 로고    scopus 로고
    • Familial hypercholester-olemia and atherosclerosis in cloned minipigs created by DNA transposition of a human PCSK9 gain-of-function mutant
    • Al-Mashhadi RH, Sørensen CB, Kragh PM, et al. Familial hypercholester-olemia and atherosclerosis in cloned minipigs created by DNA transposition of a human PCSK9 gain-of-function mutant. Sci Transl Med. 2013;5:166ra1.
    • (2013) Sci Transl Med. , vol.5 , pp. 166ra1
    • Al-Mashhadi, R.H.1    Sørensen, C.B.2    Kragh, P.M.3
  • 29
    • 69249245353 scopus 로고    scopus 로고
    • Cellular and secreted proprotein convertase subtilisin/kexin type 9 catalytic activity in hepatocytes
    • Kourimate S, Chétiveaux M, Jarnoux AL, Lalanne F, Costet P. Cellular and secreted proprotein convertase subtilisin/kexin type 9 catalytic activity in hepatocytes. Atherosclerosis. 2009;206:134-140.
    • (2009) Atherosclerosis. , vol.206 , pp. 134-140
    • Kourimate, S.1    Chétiveaux, M.2    Jarnoux, A.L.3    Lalanne, F.4    Costet, P.5
  • 30
    • 0034202522 scopus 로고    scopus 로고
    • Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro
    • Miao CH, Ohashi K, Patijn GA, Meuse L, Ye X, Thompson AR, Kay MA. Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Mol Ther. 2000;1:522-532.
    • (2000) Mol Ther. , vol.1 , pp. 522-532
    • Miao, C.H.1    Ohashi, K.2    Patijn, G.A.3    Meuse, L.4    Ye, X.5    Thompson, A.R.6    Kay, M.A.7
  • 33
    • 0028829459 scopus 로고
    • Variability in cholesterol content in serum and aortic tissue in apolipoprotein E-defcient mice is comparable in inbred (129/Sv) and outbred (mixed 129/Sv and C57BL/6) mice
    • van Ree JH, van den Broek WJ, Dahlmans VE, Wieringa B, Frants RR, Havekes LM, Hofker MH. Variability in cholesterol content in serum and aortic tissue in apolipoprotein E-defcient mice is comparable in inbred (129/Sv) and outbred (mixed 129/Sv and C57BL/6) mice. Atherosclerosis. 1995;118:165-167.
    • (1995) Atherosclerosis. , vol.118 , pp. 165-167
    • Van Ree, J.H.1    Van Den Broek, W.J.2    Dahlmans, V.E.3    Wieringa, B.4    Frants, R.R.5    Havekes, L.M.6    Hofker, M.H.7
  • 35
    • 0028345073 scopus 로고
    • Massive xan-thomatosis and atherosclerosis in cholesterol-fed low density lipoprotein receptor-negative mice
    • Ishibashi S, Goldstein JL, Brown MS, Herz J, Burns DK. Massive xan-thomatosis and atherosclerosis in cholesterol-fed low density lipoprotein receptor-negative mice. J Clin Invest. 1994;93:1885-1893.
    • (1994) J Clin Invest. , vol.93 , pp. 1885-1893
    • Ishibashi, S.1    Goldstein, J.L.2    Brown, M.S.3    Herz, J.4    Burns, D.K.5
  • 37
    • 84867088984 scopus 로고    scopus 로고
    • Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model
    • Suhy DA, Kao SC, Mao T, et al. Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model. Mol Ther. 2012;20:1737-1749.
    • (2012) Mol Ther. , vol.20 , pp. 1737-1749
    • Suhy, D.A.1    Kao, S.C.2    Mao, T.3
  • 38
    • 0141636324 scopus 로고    scopus 로고
    • Adeno-associated viral vectors for gene transfer and gene therapy
    • Büeler H. Adeno-associated viral vectors for gene transfer and gene therapy. Biol Chem. 1999;380:613-622.
    • (1999) Biol Chem. , vol.380 , pp. 613-622
    • Büeler, H.1
  • 40
  • 41
    • 0029115499 scopus 로고
    • Normal plasma lipoproteins and fertility in gene-targeted mice homozygous for a disruption in the gene encoding very low density lipoprotein receptor
    • Frykman PK, Brown MS, Yamamoto T, Goldstein JL, 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. 1995;92:8453-8457.
    • (1995) Proc Natl Acad Sci USA. , vol.92 , pp. 8453-8457
    • Frykman, P.K.1    Brown, M.S.2    Yamamoto, T.3    Goldstein, J.L.4    Herz, J.5
  • 43
    • 0034521695 scopus 로고    scopus 로고
    • LDL receptor defciency unmasks altered VLDL triglyceride metabolism in VLDL receptor transgenic and knockout mice
    • Tacken PJ, Teusink B, Jong MC, Harats D, Havekes LM, van Dijk KW, Hofker MH. LDL receptor defciency unmasks altered VLDL triglyceride metabolism in VLDL receptor transgenic and knockout mice. J Lipid Res. 2000;41:2055-2062.
    • (2000) J Lipid Res. , vol.41 , pp. 2055-2062
    • Tacken, P.J.1    Teusink, B.2    Jong, M.C.3    Harats, D.4    Havekes, L.M.5    Van Dijk, K.W.6    Hofker, M.H.7
  • 44
    • 33644820684 scopus 로고    scopus 로고
    • Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    • Manno CS, Pierce GF, Arruda VR, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med. 2006;12:342-347.
    • (2006) Nat Med. , vol.12 , pp. 342-347
    • Manno, C.S.1    Pierce, G.F.2    Arruda, V.R.3
  • 47
    • 84876801739 scopus 로고    scopus 로고
    • The safety of therapeutic monoclonal antibodies: Implications for cardiovascular disease and targeting the PCSK9 pathway
    • Catapano AL, Papadopoulos N. The safety of therapeutic monoclonal antibodies: implications for cardiovascular disease and targeting the PCSK9 pathway. Atherosclerosis. 2013;228:18-28.
    • (2013) Atherosclerosis. , vol.228 , pp. 18-28
    • Catapano, A.L.1    Papadopoulos, N.2
  • 49
    • 84863494422 scopus 로고    scopus 로고
    • Effect of a monoclonal antibody to PCSK9, REGN727/SAR236553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezeti-mibe therapy: A phase 2 randomised controlled trial
    • Stein EA, Gipe D, Bergeron J, Gaudet D, Weiss R, Dufour R, Wu R, Pordy R. Effect of a monoclonal antibody to PCSK9, REGN727/SAR236553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezeti-mibe therapy: a phase 2 randomised controlled trial. Lancet. 2012;380:29-36.
    • (2012) Lancet. , vol.380 , pp. 29-36
    • Stein, E.A.1    Gipe, D.2    Bergeron, J.3    Gaudet, D.4    Weiss, R.5    Dufour, R.6    Wu, R.7    Pordy, R.8


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