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

被引：87

作者：

Gaudet, Daniel ^{[1
]}

de Wal, Janneke ^{[2
]}

Tremblay, Karine ^{[1
]}

Dery, Stephane ^{[1
]}

van Deventer, Sander ^{[2
]}

Freidig, Andreas ^{[2
]}

Brisson, Diane ^{[1
]}

Methot, Julie ^{[1
]}

机构：

[1] Univ Montreal, Dept Med, ECOGENE Clin Res Ctr 21, Chicoutimi Hosp, Chicoutimi, PQ G7H 7P2, Canada

[2] Amsterdam Mol Therapeut AMT BV, Amsterdam, Netherlands

来源：

ATHEROSCLEROSIS SUPPLEMENTS | 2010年 / 11卷 / 01期

基金：

加拿大健康研究院;

关键词：

Lipoprotein lipase; Gene therapy; Pancreatitis; Chylomicronaemia; RENAL-TRANSPLANT RECIPIENTS; CHRONIC-PANCREATITIS; MYCOPHENOLATE-MOFETIL; RANDOMIZED-TRIAL; CYCLOSPORINE; MUTATIONS; RISK; MULTICENTER; COMBINATION;

D O I：

10.1016/j.atherosclerosissup.2010.03.004

中图分类号：

R6 [外科学];

学科分类号：

1002 ; 100210 ;

摘要：

Alipogene tiparvovec (AAV1-LPLS447X) gene therapy is developed to prevent complications and decrease the clinical morbidity of lipoprotein lipase deficiency (LPLD). LPLD is an autosomal recessive disease associated with severe hypertriglyceridemia (hyperTG), severe chylomicronaemia, and low HDL. Acute pancreatitis, the most frequent serious clinical LPLD complication, is a complex and heterogeneous inflammatory condition having many causes including hyperTG and chylomicronaemia. In many patients, low fat diet and currently available lipid lowering drugs are ineffective to prevent hyperTG or pancreatitis in LPLD. The clinical development program of alipogene tiparvovec includes observational studies as well as phase I/II and II/III clinical trials. Pooled data are collected on safety and efficacy issues, including the incidence of pancreatitis. (C) 2010 Elsevier Ireland Ltd. All rights reserved.

引用

页码：55 / 60

页数：6

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