From Mutation Identification to Therapy: Discovery and Origins of the First Approved Gene Therapy in the Western World

被引:29
作者
Kastelein, John J. P. [1 ]
Ross, Colin J. D. [2 ,3 ,4 ]
Hayden, Michael R. [3 ,4 ]
机构
[1] Univ Amsterdam, Acad Med Ctr, Dept Vasc Med, NL-1105 AZ Amsterdam, Netherlands
[2] Univ British Columbia, Dept Pediat, Vancouver, BC V5Z 4H4, Canada
[3] Univ British Columbia, Ctr Mol Med & Therapeut, Child & Family Res Inst, Vancouver, BC V5Z 4H4, Canada
[4] Univ British Columbia, Dept Med Genet, Vancouver, BC V5Z 4H4, Canada
关键词
LIPOPROTEIN-LIPASE GENE; CORONARY-ARTERY-DISEASE; LPLS447X BENEFICIAL MUTATION; FRENCH-CANADIAN POPULATION; HDL CHOLESTEROL LEVELS; GUINEA-PIG HEARTS; FAMILIAL HYPERCHOLESTEROLEMIA; CAUSES CHYLOMICRONEMIA; PLASMA-LIPOPROTEINS; DEFICIENT PATIENTS;
D O I
10.1089/hum.2013.063
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
引用
收藏
页码:472 / 478
页数:7
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共 64 条
  • [11] DIONNE C, 1992, ANN GENET-PARIS, V35, P89
  • [12] Severe hypertriglyceridemia and pancreatitis: presentation and management
    Ewald, Nils
    Hardt, Philip D.
    Kloer, Hans-Ulrich
    [J]. CURRENT OPINION IN LIPIDOLOGY, 2009, 20 (06) : 497 - 504
  • [13] Correction of hypertriglyceridemia and impaired fat tolerance in lipoprotein lipase-deficient mice by adenovirus-mediated expression of human lipoprotein lipase
    Excoffon, KJDA
    Liu, GQ
    Miao, L
    Wilson, JE
    McManus, BM
    Semenkovich, CF
    Coleman, T
    Benoit, P
    Duverger, N
    Branellec, D
    Denefle, P
    Hayden, MR
    Lewis, MES
    [J]. ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY, 1997, 17 (11) : 2532 - 2539
  • [14] Assessment of French patients with LPL deficiency for French Canadian mutations
    Foubert, L
    DeGennes, JL
    Lagarde, JP
    Ehrenborg, E
    Raisonnier, A
    Girardet, JP
    Hayden, MR
    Benlian, P
    [J]. JOURNAL OF MEDICAL GENETICS, 1997, 34 (08) : 672 - 675
  • [15] Foubert L, 1997, HUM MUTAT, V10, P179
  • [16] GENE THERAPY FOR HUMAN GENETIC DISEASE
    FRIEDMANN, T
    ROBLIN, R
    [J]. SCIENCE, 1972, 175 (4025) : 949 - +
  • [17] A common truncation variant of lipoprotein lipase (Ser447X) confers protection against coronary heart disease:: the Framingham Offspring Study
    Gagné, SE
    Larson, MG
    Pimstone, SN
    Schaefer, EJ
    Kastelein, JJP
    Wilson, PWF
    Ordovas, JM
    Hayden, MR
    [J]. CLINICAL GENETICS, 1999, 55 (06) : 450 - 454
  • [18] Galton D J, 1996, Z Gastroenterol, V34 Suppl 3, P56
  • [19] Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
    Gao, GP
    Alvira, MR
    Wang, LL
    Calcedo, R
    Johnston, J
    Wilson, JM
    [J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2002, 99 (18) : 11854 - 11859
  • [20] Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
    Gaudet, D.
    Methot, J.
    Dery, S.
    Brisson, D.
    Essiembre, C.
    Tremblay, G.
    Tremblay, K.
    de Wal, J.
    Twisk, J.
    van den Bulk, N.
    Sier-Ferreira, V.
    van Deventer, S.
    [J]. GENE THERAPY, 2013, 20 (04) : 361 - 369