메뉴 건너뛰기




Volumn 11, Issue 1, 2010, Pages 55-60

Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency

Author keywords

Chylomicronaemia; Gene therapy; Lipoprotein lipase; Pancreatitis

Indexed keywords

ALIPOGENE TIPARVOVEC; AMT 010; AMT 011; ANTILIPEMIC AGENT; CYCLOSPORIN; GENE THERAPY AGENT; GLYBERA; HIGH DENSITY LIPOPROTEIN; METHYLPREDNISOLONE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

EID: 77952571307     PISSN: 15675688     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.atherosclerosissup.2010.03.004     Document Type: Article
Times cited : (108)

References (33)
  • 1
    • 0002316655 scopus 로고    scopus 로고
    • Familial lipoprotein lipase deficiency, Apo C-II deficiency and hepatic lipase deficiency
    • Scriver C.R., Beaudet A.L., Sly W.S., and Valle D. (Eds), McGraw-Hill, New York
    • Brunzell J.D., and Deeb S.S. Familial lipoprotein lipase deficiency, Apo C-II deficiency and hepatic lipase deficiency. In: Scriver C.R., Beaudet A.L., Sly W.S., and Valle D. (Eds). The metabolic basis of inherited disease. 8th ed. (2000), McGraw-Hill, New York 2789-2816
    • (2000) The metabolic basis of inherited disease. 8th ed. , pp. 2789-2816
    • Brunzell, J.D.1    Deeb, S.S.2
  • 2
    • 0035292694 scopus 로고    scopus 로고
    • Lipoprotein lipase: physiology, biochemistry, and molecular biology
    • Goldberg I.J., and Merkel M. Lipoprotein lipase: physiology, biochemistry, and molecular biology. Front Biosci 6 (2001) D388-405
    • (2001) Front Biosci , vol.6
    • Goldberg, I.J.1    Merkel, M.2
  • 3
    • 0024533690 scopus 로고
    • Primary lipoprotein-lipase-activity deficiency: clinical investigation of a French Canadian population
    • Gagne C., Brun L.D., Julien P., et al. Primary lipoprotein-lipase-activity deficiency: clinical investigation of a French Canadian population. CMAJ 140 (1989) 405-411
    • (1989) CMAJ , vol.140 , pp. 405-411
    • Gagne, C.1    Brun, L.D.2    Julien, P.3
  • 4
    • 77952575107 scopus 로고    scopus 로고
    • Chylomicronemia with low postheparin lipoprotein lipase levels in the setting of GPIHBP1 defects
    • Franssen R., Young S.G., Peelman F., et al. Chylomicronemia with low postheparin lipoprotein lipase levels in the setting of GPIHBP1 defects. Circ Cardiovasc Genet (2010)
    • (2010) Circ Cardiovasc Genet
    • Franssen, R.1    Young, S.G.2    Peelman, F.3
  • 5
    • 23044440065 scopus 로고    scopus 로고
    • Combination of circulating antilipoprotein lipase (Anti-LPL) antibody and heterozygous S172 fsX179 mutation of LPL gene leading to chronic hyperchylomicronemia
    • Pruneta-Deloche V., Marcais C., Perrot L., et al. Combination of circulating antilipoprotein lipase (Anti-LPL) antibody and heterozygous S172 fsX179 mutation of LPL gene leading to chronic hyperchylomicronemia. J Clin Endocrinol Metab 90 (2005) 3995-3998
    • (2005) J Clin Endocrinol Metab , vol.90 , pp. 3995-3998
    • Pruneta-Deloche, V.1    Marcais, C.2    Perrot, L.3
  • 6
    • 0027455653 scopus 로고
    • Dietary treatment and growth of hyperchylomicronemic children severely restricted in dietary fat
    • Black D.M., and Sprecher D.L. Dietary treatment and growth of hyperchylomicronemic children severely restricted in dietary fat. Am J Dis Child 147 (1993) 60-62
    • (1993) Am J Dis Child , vol.147 , pp. 60-62
    • Black, D.M.1    Sprecher, D.L.2
  • 9
    • 0035109764 scopus 로고    scopus 로고
    • Chronic pancreatitis: diagnosis, classification, and new genetic developments
    • Etemad B., and Whitcomb D.C. Chronic pancreatitis: diagnosis, classification, and new genetic developments. Gastroenterology 120 (2001) 682-707
    • (2001) Gastroenterology , vol.120 , pp. 682-707
    • Etemad, B.1    Whitcomb, D.C.2
  • 10
    • 0029808302 scopus 로고    scopus 로고
    • Premature atherosclerosis in patients with familial chylomicronemia caused by mutations in the lipoprotein lipase gene
    • Benlian P., De Gennes J.L., Foubert L., et al. Premature atherosclerosis in patients with familial chylomicronemia caused by mutations in the lipoprotein lipase gene. N Engl J Med 335 (1996) 848-854
    • (1996) N Engl J Med , vol.335 , pp. 848-854
    • Benlian, P.1    De Gennes, J.L.2    Foubert, L.3
  • 12
    • 70350225542 scopus 로고    scopus 로고
    • Chronic pancreatitis: genetics and pathogenesis
    • Chen J.M., and Ferec C. Chronic pancreatitis: genetics and pathogenesis. Annu Rev Genomics Hum Genet 10 (2009) 63-87
    • (2009) Annu Rev Genomics Hum Genet , vol.10 , pp. 63-87
    • Chen, J.M.1    Ferec, C.2
  • 13
    • 4544322709 scopus 로고    scopus 로고
    • Clinical characterization of patients with idiopathic chronic pancreatitis and SPINK1 mutations
    • Schneider A., Barmada M.M., Slivka A., et al. Clinical characterization of patients with idiopathic chronic pancreatitis and SPINK1 mutations. Scand J Gastroenterol 39 (2004) 903-904
    • (2004) Scand J Gastroenterol , vol.39 , pp. 903-904
    • Schneider, A.1    Barmada, M.M.2    Slivka, A.3
  • 14
    • 0032480346 scopus 로고    scopus 로고
    • Relation between mutations of the cystic fibrosis gene and idiopathic pancreatitis
    • Cohn J.A., Friedman K.J., Noone P.G., et al. Relation between mutations of the cystic fibrosis gene and idiopathic pancreatitis. N Engl J Med 339 (1998) 653-658
    • (1998) N Engl J Med , vol.339 , pp. 653-658
    • Cohn, J.A.1    Friedman, K.J.2    Noone, P.G.3
  • 15
    • 77951920963 scopus 로고    scopus 로고
    • Genetic aspects of pancreatitis
    • Whitcomb D.C. Genetic aspects of pancreatitis. Annu Rev Med 61 (2010) 413-424
    • (2010) Annu Rev Med , vol.61 , pp. 413-424
    • Whitcomb, D.C.1
  • 16
    • 35348853246 scopus 로고    scopus 로고
    • The pancreas misled: signals to pancreatitis
    • Criddle D.N., McLaughlin E., Murphy J.A., et al. The pancreas misled: signals to pancreatitis. Pancreatology 7 (2007) 436-446
    • (2007) Pancreatology , vol.7 , pp. 436-446
    • Criddle, D.N.1    McLaughlin, E.2    Murphy, J.A.3
  • 17
    • 56049087543 scopus 로고    scopus 로고
    • Investigation and management of hypertriglyceridaemia
    • Ferns G., Keti V., and Griffin B. Investigation and management of hypertriglyceridaemia. J Clin Pathol 61 (2008) 1174-1183
    • (2008) J Clin Pathol , vol.61 , pp. 1174-1183
    • Ferns, G.1    Keti, V.2    Griffin, B.3
  • 18
    • 0026479458 scopus 로고
    • Chylomicronemia syndrome
    • Chait A., and Brunzell J.D. Chylomicronemia syndrome. Adv Intern Med 37 (1992) 249-273
    • (1992) Adv Intern Med , vol.37 , pp. 249-273
    • Chait, A.1    Brunzell, J.D.2
  • 19
    • 77952559247 scopus 로고    scopus 로고
    • Paradis G. Effect of a nutritional supplement rich containing medium-chain triglyceride (MCT) and the adjunction of fibrates on lipid-lipoprotein profile in subjects with homozygous LPL deficiency. Master of Sciences Thesis, Université Laval, Québec; 2001.
    • Paradis G. Effect of a nutritional supplement rich containing medium-chain triglyceride (MCT) and the adjunction of fibrates on lipid-lipoprotein profile in subjects with homozygous LPL deficiency. Master of Sciences Thesis, Université Laval, Québec; 2001.
  • 20
    • 33749451873 scopus 로고    scopus 로고
    • AAV1-LPL(S447X) gene therapy reduces hypertriglyceridemia in apoE2 knock in mice
    • Rip J., van Dijk K.W., Sierts J.A., et al. AAV1-LPL(S447X) gene therapy reduces hypertriglyceridemia in apoE2 knock in mice. Biochim Biophys Acta 1761 (2006) 1163-1168
    • (2006) Biochim Biophys Acta , vol.1761 , pp. 1163-1168
    • Rip, J.1    van Dijk, K.W.2    Sierts, J.A.3
  • 21
    • 0033536024 scopus 로고    scopus 로고
    • Lipoprotein lipase mutations, plasma lipids and lipoproteins, and risk of ischemic heart disease. A meta-analysis
    • Wittrup H.H., Tybjaerg-Hansen A., and Nordestgaard B.G. Lipoprotein lipase mutations, plasma lipids and lipoproteins, and risk of ischemic heart disease. A meta-analysis. Circulation 99 (1999) 2901-2907
    • (1999) Circulation , vol.99 , pp. 2901-2907
    • Wittrup, H.H.1    Tybjaerg-Hansen, A.2    Nordestgaard, B.G.3
  • 22
    • 34447637932 scopus 로고    scopus 로고
    • Presence of lipoprotein lipase S447X stop codon affects the magnitude of interleukin 8 release after cardiac surgery with cardiopulmonary bypass
    • Ak K., Isbir S., Tekeli A., et al. Presence of lipoprotein lipase S447X stop codon affects the magnitude of interleukin 8 release after cardiac surgery with cardiopulmonary bypass. J Thorac Cardiovasc Surg 134 (2007) 477-483
    • (2007) J Thorac Cardiovasc Surg , vol.134 , pp. 477-483
    • Ak, K.1    Isbir, S.2    Tekeli, A.3
  • 23
    • 57549090220 scopus 로고    scopus 로고
    • Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients
    • Stroes E.S., Nierman M.C., Meulenberg J.J., et al. Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arterioscler Thromb Vasc Biol 28 (2008) 2303-2304
    • (2008) Arterioscler Thromb Vasc Biol , vol.28 , pp. 2303-2304
    • Stroes, E.S.1    Nierman, M.C.2    Meulenberg, J.J.3
  • 24
    • 27744446128 scopus 로고    scopus 로고
    • Gene therapy for lipoprotein lipase deficiency: working toward clinical application
    • Rip J., Nierman M.C., Sierts J.A., et al. Gene therapy for lipoprotein lipase deficiency: working toward clinical application. Hum Gene Ther 16 (2005) 1276-1286
    • (2005) Hum Gene Ther , vol.16 , pp. 1276-1286
    • Rip, J.1    Nierman, M.C.2    Sierts, J.A.3
  • 25
    • 69949160511 scopus 로고    scopus 로고
    • AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells
    • Mingozzi F., Meulenberg J.J., Hui D.J., et al. AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells. Blood 114 (2009) 2077-2086
    • (2009) Blood , vol.114 , pp. 2077-2086
    • Mingozzi, F.1    Meulenberg, J.J.2    Hui, D.J.3
  • 26
    • 33750033625 scopus 로고    scopus 로고
    • Impact of cyclosporine reduction with MMF: a randomized trial in chronic allograft dysfunction. The 'reference' study
    • Frimat L., Cassuto-Viguier E., Charpentier B., et al. Impact of cyclosporine reduction with MMF: a randomized trial in chronic allograft dysfunction. The 'reference' study. Am J Transplant 6 (2006) 2725-2734
    • (2006) Am J Transplant , vol.6 , pp. 2725-2734
    • Frimat, L.1    Cassuto-Viguier, E.2    Charpentier, B.3
  • 27
    • 33745200700 scopus 로고    scopus 로고
    • Efficacy and safety of enteric-coated mycophenolate sodium (myfortic) in de novo renal transplant recipients: results of a 12-month multicenter, open-label, prospective study
    • 1301-1306.26
    • Vogt B., Antoniadis A., Klinger M., and Vitko S. Efficacy and safety of enteric-coated mycophenolate sodium (myfortic) in de novo renal transplant recipients: results of a 12-month multicenter, open-label, prospective study. Transplant Proc 38 (2006) 1301-1306.26
    • (2006) Transplant Proc , vol.38
    • Vogt, B.1    Antoniadis, A.2    Klinger, M.3    Vitko, S.4
  • 28
    • 0037183617 scopus 로고    scopus 로고
    • Short-term combination of mycophenolate mofetil with cyclosporine as a therapeutic option for renal transplant recipients: a prospective, multicenter, randomized study
    • Sadek S., Medina J., Arias M., et al. Short-term combination of mycophenolate mofetil with cyclosporine as a therapeutic option for renal transplant recipients: a prospective, multicenter, randomized study. Transplantation 74 (2002) 511-517
    • (2002) Transplantation , vol.74 , pp. 511-517
    • Sadek, S.1    Medina, J.2    Arias, M.3
  • 29
    • 0034905252 scopus 로고    scopus 로고
    • Randomized trial of tacrolimus plus mycophenolate mofetil or azathioprine versus cyclosporine oral solution (modified) plus mycophenolate mofetil after cadaveric kidney transplantation: results at 2 years
    • Ahsan N., Johnson C., Gonwa T., et al. Randomized trial of tacrolimus plus mycophenolate mofetil or azathioprine versus cyclosporine oral solution (modified) plus mycophenolate mofetil after cadaveric kidney transplantation: results at 2 years. Transplantation 72 (2001) 245-250
    • (2001) Transplantation , vol.72 , pp. 245-250
    • Ahsan, N.1    Johnson, C.2    Gonwa, T.3
  • 31
    • 11844303500 scopus 로고    scopus 로고
    • Recent developments in gene transfer: risk and ethics
    • Kimmelman J. Recent developments in gene transfer: risk and ethics. BMJ 330 (2005) 79-82
    • (2005) BMJ , vol.330 , pp. 79-82
    • Kimmelman, J.1
  • 32
    • 70449527808 scopus 로고    scopus 로고
    • Accepting risk in clinical research: is the gene therapy field becoming too risk-averse?
    • Deakin C.T., Alexander I.E., and Kerridge I. Accepting risk in clinical research: is the gene therapy field becoming too risk-averse?. Mol Ther 17 (2009) 1842-1848
    • (2009) Mol Ther , vol.17 , pp. 1842-1848
    • Deakin, C.T.1    Alexander, I.E.2    Kerridge, I.3
  • 33
    • 0019540987 scopus 로고
    • On the quantitative definition of risk
    • Kaplan S., and Garrick B.J. On the quantitative definition of risk. Risk Anal 1 (1981) 11-27
    • (1981) Risk Anal , vol.1 , pp. 11-27
    • Kaplan, S.1    Garrick, B.J.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.