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

Authors: Gaudet, Daniel; de Wal, Janneke; Tremblay, Karine; Déry, Stéphane; van Deventer, Sander; Freidig, Andreas; Brisson, Diane.Méthot, Julie
Abstract: 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.
Document Type: Article de recherche
Issue Date: 27 April 2010
Open Access Date: Restricted access
Document version: VoR
This document was published in: Atherosclerosis. Supplement, Vol. 11, 55-60 (2010)
Elsevier Science
Alternative version: 10.1016/j.atherosclerosissup.2010.03.004
Collection:Articles publiés dans des revues avec comité de lecture

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