메뉴 건너뛰기




Volumn 11, Issue 6, 2009, Pages 681-691

Alipogene tiparvovec, an adeno-associated virus encoding the Ser 447X variant of the human lipoprotein lipase gene for the treatment of patients with lipoprotein lipase deficiency

Author keywords

[No Author keywords available]

Indexed keywords

ALIPOGENE TIPARVOVEC; GLYBERA; HIGH DENSITY LIPOPROTEIN CHOLESTEROL; LIPOPROTEIN LIPASE; PROTEIN; SERINE; TRIACYLGLYCEROL; UNCLASSIFIED DRUG; VIRUS VECTOR;

EID: 72449169616     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review
Times cited : (44)

References (62)
  • 1
    • 72449125652 scopus 로고    scopus 로고
    • Amsterdam Molecular Therapeutics strengthens lipoprotein lipase gene therapy program
    • BV PRESS RELEASE. August 20
    • Amsterdam Molecular Therapeutics strengthens lipoprotein lipase gene therapy program. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2001 August 20
    • (2001) Amsterdam Molecular Therapeutics
  • 3
    • 0028819754 scopus 로고
    • Severe hypertriglyceridemia, reduced high density lipoprotein, and neonatal death in lipoprotein lipase knockout mice. Mild hypertriglyceridemia with impaired very low density lipoprotein clearance in heterozygotes
    • Severe hypertriglyceridemia, reduced high density lipoprotein, and neonatal death in lipoprotein lipase knockout mice. Mild hypertriglyceridemia with impaired very low density lipoprotein clearance in heterozygotes. Weinstock PH, Bisgaier CL, Aalto-Setala K, Radner H, Ramakrishnan R, Levak-Frank S, Essenburg AD, Zechner R, Breslow JL J CLIN INVEST 1995 96 6 2555-2568
    • (1995) Breslow JL J CLIN INVEST , vol.96 , Issue.6 , pp. 2555-2568
    • Weinstock, P.H.1    Bisgaier, C.L.2    Aalto-Setala, K.3    Radner, H.4    Ramakrishnan, R.5    Levak-Frank, S.6    Essenburg, A.D.7    Zechner, R.8
  • 4
    • 72449180389 scopus 로고    scopus 로고
    • AMT obtains Orphan Drug designation for its LPL gene therapy product
    • BV PRESS RELEASE. March 17
    • AMT obtains Orphan Drug designation for its LPL gene therapy product. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2004 March 17
    • (2004) Amsterdam Molecular Therapeutics
  • 5
    • 72449145793 scopus 로고    scopus 로고
    • Amsterdam Molecular Therapeutics BV (AMT) acquires license to AAV patents from Targeted Genetics Corporation
    • BV PRESS RELEASE. December 12
    • Amsterdam Molecular Therapeutics BV (AMT) acquires license to AAV patents from Targeted Genetics Corporation. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2006 December 12
    • (2006) Amsterdam Molecular Therapeutics
  • 6
    • 72449138107 scopus 로고    scopus 로고
    • Amsterdam Molecular Therapeutics announces intention to launch an Initial Public Offering (IPO) on Euronext Amsterdam
    • BV PRESS RELEASE. May 22
    • Amsterdam Molecular Therapeutics announces intention to launch an Initial Public Offering (IPO) on Euronext Amsterdam. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2007 May 22
    • (2007) Amsterdam Molecular Therapeutics
  • 7
    • 72449176892 scopus 로고    scopus 로고
    • AMT presents positive results of phase I/II study with lead product at annual meeting of American Society of Gene Therapy
    • BV PRESS RELEASE. May 31
    • AMT presents positive results of phase I/II study with lead product at annual meeting of American Society of Gene Therapy. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2007 May 31
    • (2007) Amsterdam Molecular Therapeutics
  • 8
    • 72449183138 scopus 로고    scopus 로고
    • AMT receives Orphan Drug designation from FDA for AMT-011 for the treatment of lipoprotein lipase deficiency
    • BV PRESS RELEASE. June 04
    • AMT receives Orphan Drug designation from FDA for AMT-011 for the treatment of lipoprotein lipase deficiency. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2007 June 04
    • (2007) Amsterdam Molecular Therapeutics
  • 9
    • 72449183626 scopus 로고    scopus 로고
    • AMT announces positive interim clinical data on its lead product AMT-011
    • BV PRESS RELEASE. May 30
    • AMT announces positive interim clinical data on its lead product AMT-011. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2008 May 30
    • (2008) Amsterdam Molecular Therapeutics
  • 10
    • 72449167444 scopus 로고    scopus 로고
    • AMT intends to incorporate additional data to its marketing authorization dossier for Glybera
    • BV PRESS RELEASE. October 03
    • AMT intends to incorporate additional data to its marketing authorization dossier for Glybera. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2008 October 03
    • (2008) Amsterdam Molecular Therapeutics
  • 11
    • 72449177883 scopus 로고    scopus 로고
    • 15-17 October 2008. European Medicines Agency PRESS RELEASE 2008 October 24
    • Meeting highlights from the Paediatric Committee, 15-17 October 2008. European Medicines Agency PRESS RELEASE 2008 October 24
    • Meeting Highlights from the Paediatric Committee
  • 12
    • 72449132351 scopus 로고    scopus 로고
    • AMT: Glybera lowers pancreatitis incidence significantly
    • BV PRESS RELEASE. January 28
    • AMT: Glybera lowers pancreatitis incidence significantly. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2009 January 28
    • (2009) Amsterdam Molecular Therapeutics
  • 13
    • 72449137149 scopus 로고    scopus 로고
    • AMT starts preregistration trial for Glybera
    • BV PRESS RELEASE. May 07
    • AMT starts preregistration trial for Glybera. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2009 May 07
    • (2009) Amsterdam Molecular Therapeutics
  • 15
  • 19
    • 72449122333 scopus 로고    scopus 로고
    • Glybera shows long-term health benefits
    • BV PRESS RELEASE. June 18
    • Glybera shows long-term health benefits. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2009 June 18
    • (2009) Amsterdam Molecular Therapeutics
  • 24
    • 72449123222 scopus 로고    scopus 로고
    • Treatment of female mice with AAV1-LPLS447X at 4 weeks prior to mating has no effect on reproductive potential and does not result in embryotoxicity or germ line transmission
    • Or 51
    • Treatment of female mice with AAV1-LPLS447X at 4 weeks prior to mating has no effect on reproductive potential and does not result in embryotoxicity or germ line transmission. Van Amersfoort E, Waalkens-Berendsen I, Bauer U, Meulenberg J, Twisk J HUM GENE THER 2007 18 10 Or 51
    • (2007) Twisk J HUM GENE THER , vol.18 , Issue.10
    • Van Amersfoort, E.1    Waalkens-Berendsen, I.2    Bauer, U.3    Meulenberg, J.4
  • 26
    • 0028042106 scopus 로고
    • Lipoprotein-lipase - Structure, function and mechanism of action
    • Lipoprotein-lipase - Structure, function and mechanism of action. Santamarina-Fojo S, Brewer HB Jr INT J CLIN LAB RES 1994 24 3 143-147
    • (1994) INT J CLIN LAB RES , vol.24 , Issue.3 , pp. 143-147
    • Santamarina-Fojo, S.1    Brewer Jr., H.B.2
  • 28
    • 0024533690 scopus 로고
    • Primary lipoprotein-lipase-activity deficiency: Clinical investigation of a French-Canadian population
    • Primary lipoprotein-lipase-activity deficiency: Clinical investigation of a French-Canadian population. Gagne C, Brun LD, Julien P, Moorjani S, Lupien PJ CMAJ 1989 140 4 405-411
    • (1989) CMAJ , vol.140 , Issue.4 , pp. 405-411
    • Gagne, C.1    Brun, L.D.2    Julien, P.3    Moorjani, S.4    Lupien, P.J.5
  • 29
    • 34247471569 scopus 로고    scopus 로고
    • Hypertriglyceridemia: Its etiology, effects and treatment
    • Hypertriglyceridemia: Its etiology, effects and treatment. Yuan G, Al-Shali KZ, Hegele RA CMAJ 2007 176 8 1113-1120
    • (2007) CMAJ , vol.176 , Issue.8 , pp. 1113-1120
    • Yuan, G.1    Al-Shali, K.Z.2    Hegele, R.A.3
  • 30
    • 0024397097 scopus 로고
    • Structure of the human lipoprotein lipase gene
    • Structure of the human lipoprotein lipase gene. Deeb SS, Peng RL BIOCHEMISTRY 1989 28 10 4131-4135
    • (1989) BIOCHEMISTRY , vol.28 , Issue.10 , pp. 4131-4135
    • Deeb, S.S.1    Peng, R.L.2
  • 31
    • 0036906508 scopus 로고    scopus 로고
    • Lipoprotein lipase: Genetics, lipid uptake, and regulation
    • Lipoprotein lipase: Genetics, lipid uptake, and regulation. Merkel M, Eckel RH, Goldberg IJ J LIPID RES 2002 43 12 1997-2006
    • (2002) J LIPID RES , vol.43 , Issue.12 , pp. 1997-2006
    • Merkel, M.1    Eckel, R.H.2    Goldberg, I.J.3
  • 32
    • 35448983964 scopus 로고    scopus 로고
    • Homozygous missense mutation (G56R) in glycosylphosphatidylinositol- anchored high-density lipoproteinbinding protein 1 (GPI-HBP1) in two siblings with fasting chylomicronemia (MIM 144650)
    • Homozygous missense mutation (G56R) in glycosylphosphatidylinositol- anchored high-density lipoproteinbinding protein 1 (GPI-HBP1) in two siblings with fasting chylomicronemia (MIM 144650). Wang J, Hegele RA LIPIDS HEALTH DIS 2007 6 23
    • (2007) LIPIDS HEALTH DIS , vol.6 , pp. 23
    • Wang, J.1    Hegele, R.A.2
  • 38
    • 0027296725 scopus 로고
    • Overexpression of human lipoprotein-lipase in transgenic mice. Resistance to diet-induced hypertriglyceridemia and hypercholesterolemia
    • Overexpression of human lipoprotein-lipase in transgenic mice. Resistance to diet-induced hypertriglyceridemia and hypercholesterolemia. Shimada M, Shimano H, Gotoda T, Yamamoto K, Kawamura M, Inaba T, Yazaki Y, Yamada N J BIOL CHEM 1993 268 24 17924-17929
    • (1993) J BIOL CHEM , vol.268 , Issue.24 , pp. 17924-17929
    • Shimada, M.1    Shimano, H.2    Gotoda, T.3    Yamamoto, K.4    Kawamura, M.5    Inaba, T.6    Yazaki, Y.7    Yamada, N.8
  • 39
    • 0030002205 scopus 로고    scopus 로고
    • Suppression of diet-induced atherosclerosis in low density lipoprotein receptor knockout mice overexpressing lipoprotein lipase
    • Suppression of diet-induced atherosclerosis in low density lipoprotein receptor knockout mice overexpressing lipoprotein lipase. Shimada M, Ishibashi S, Inaba T, Yagyu H, Harada K, Osuga J, Ohashi K, Yazaki Y, Yamada N PROC NATL ACAD SCI USA 1996 93 14 7242-7246
    • (1996) PROC NATL ACAD SCI USA , vol.93 , Issue.14 , pp. 7242-7246
    • Shimada, M.1    Ishibashi, S.2    Inaba, T.3    Yagyu, H.4    Harada, K.5    Osuga, J.6    Ohashi, K.7    Yazaki, Y.8    Yamada, N.9
  • 42
    • 72449158701 scopus 로고    scopus 로고
    • An open label, dose escalation study to assess the safety and efficacy of AAV1-LPL gene therapy with alipogene tiparvovec (AMT-011) for patients with severe hypertriglyceridemia due to lipoprotein lipase deficiency (LPLD)
    • Abs 554
    • An open label, dose escalation study to assess the safety and efficacy of AAV1-LPL gene therapy with alipogene tiparvovec (AMT-011) for patients with severe hypertriglyceridemia due to lipoprotein lipase deficiency (LPLD). Gaudet D, Brisson D, Methot J, van Deventer S ATHEROSCLEROSIS SUPPL 2009 10 2 Abs 554
    • (2009) ATHEROSCLEROSIS SUPPL , vol.10 , Issue.2
    • Gaudet, D.1    Brisson, D.2    Methot, J.3    Van Deventer, S.4
  • 46
    • 72449180388 scopus 로고    scopus 로고
    • Treatment of female mice with AAV1-LPLS447X at 4 weeks prior to mating has no effect on reproductive potential and does not result in embryotoxicity or germ line transmission
    • Abs 791
    • Treatment of female mice with AAV1-LPLS447X at 4 weeks prior to mating has no effect on reproductive potential and does not result in embryotoxicity or germ line transmission. Van Amersfoort ES, Waalkens-Berendsen IH, Bauer U, Sijtsma J, Meulenberg JJM, Twisk J AM SOC GENE THER 2008 11 Abs 791
    • (2008) AM SOC GENE THER , vol.11
    • Van Amersfoort, E.S.1    Waalkens-Berendsen, I.H.2    Bauer, U.3    Sijtsma, J.4    Meulenberg, J.J.M.5    Twisk, J.6
  • 49
    • 62649117271 scopus 로고    scopus 로고
    • Biodistribution of AAV1-LPLS447X vector co-administered with immunosuppression to lipoprotein lipase deficient patients in a phase II study
    • Abs 985
    • Biodistribution of AAV1-LPLS447X vector co-administered with immunosuppression to lipoprotein lipase deficient patients in a phase II study. Gaudet D, Methot J, Essiembre C, Brisson D, van Deventer S, Kleefstra A, Meulenberg J AM SOC GENE THER 2008 11 Abs 985
    • (2008) AM SOC GENE THER , vol.11
    • Gaudet, D.1    Methot, J.2    Essiembre, C.3    Brisson, D.4    Van Deventer, S.5    Kleefstra, A.6    Meulenberg, J.7
  • 50
  • 52
    • 72449204148 scopus 로고    scopus 로고
    • Gene transfer safety symposium: Immune responses to adeno-associated virus vectors. Session II: Immune responses in clinical studies using AAV vectors. Immune responses in a lipoprotein lipase deficiency study using AAV-1-LPLS447X. Office of Biotechnology Activities, Office of the Director, NIH US DEPARTMENT HEALTH HUM SERVICES 2007 June 19 4-5
    • Gene transfer safety symposium: Immune responses to adeno-associated virus vectors. Session II: Immune responses in clinical studies using AAV vectors. Immune responses in a lipoprotein lipase deficiency study using AAV-1-LPLS447X. Office of Biotechnology Activities, Office of the Director, NIH US DEPARTMENT HEALTH HUM SERVICES 2007 June 19 4-5
  • 54
    • 72449146759 scopus 로고    scopus 로고
    • Product pipeline
    • BV COMPANY WORLD WIDE WEB SITE. October 01
    • Product pipeline. Amsterdam Molecular Therapeutics BV COMPANY WORLD WIDE WEB SITE 2009 October 01
    • (2009) Amsterdam Molecular Therapeutics
  • 56
    • 13444274885 scopus 로고    scopus 로고
    • Gene therapy for genetic lipid disorders: Lipoprotein lipase deficiency as a paradigm
    • Gene therapy for genetic lipid disorders: Lipoprotein lipase deficiency as a paradigm. Rader DJ NETH J MED 2005 63 1 2-3
    • (2005) NETH J MED , vol.63 , Issue.1 , pp. 2-3
    • Rader, D.J.1
  • 57
    • 16044370302 scopus 로고    scopus 로고
    • An 18-year-old woman with familial chylomicronaemia who would not stick to a diet
    • An 18-year-old woman with familial chylomicronaemia who would not stick to a diet. Gasbarrini G, Mingrone G, Greco AV, Castagneto M LANCET 1996 348 9030 794
    • (1996) LANCET , vol.348 , Issue.9030 , pp. 794
    • Gasbarrini, G.1    Mingrone, G.2    Greco, A.V.3    Castagneto, M.4
  • 61
    • 72449155085 scopus 로고    scopus 로고
    • AMT provides business update for the third quarter
    • BV PRESS RELEASE 2009 November 18
    • AMT provides business update for the third quarter 2009. Amsterdam Molecular Therapeutics BV PRESS RELEASE 2009 November 18
    • (2009) Amsterdam Molecular Therapeutics
  • 62
    • 72449129495 scopus 로고    scopus 로고
    • Leading the way in gene therapy
    • BV COMPANY PRESENTATION. November
    • Leading the way in gene therapy. Amsterdam Molecular Therapeutics BV COMPANY PRESENTATION 2009 November
    • (2009) Amsterdam Molecular Therapeutics


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.