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

Gene therapy for lipoprotein lipase deficiency

(2012) Daniel Gaudet et al.   CURRENT OPINION IN LIPIDOLOGY

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

Effect of Alipogene Tiparvovec (AAV1-LPLS447X) on Postprandial Chylomicron Metabolism in Lipoprotein Lipase-Deficient Patients

(2012) André C. Carpentier et al.   JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

First gene therapy approved

(2012) Nuala Moran   NATURE BIOTECHNOLOGY

Prevalence, geographical distribution and genealogical investigations of mutation 188 of lipoprotein lipase gene in the French Canadian population of Québec

(2010) Jean Bergeron et al.   CLINICAL GENETICS

Severe hypertriglyceridemia and pancreatitis: presentation and management

(2009) Nils Ewald et al.   CURRENT OPINION IN LIPIDOLOGY

Intramuscular Administration of AAV1-Lipoprotein Lipase S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients

(2008) Erik S. Stroes et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

In vitro remodelling of plasma lipoproteins in whole plasma by lipoprotein lipase in primary and secondary hypertriglyceridaemia

(2008) E. LEVY et al.   EUROPEAN JOURNAL OF CLINICAL INVESTIGATION