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

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

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results

(2011) Terence R. Flotte et al.   HUMAN GENE THERAPY

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

(2010) Daniel Gaudet et al.   ATHEROSCLEROSIS SUPPLEMENTS

Lymphocyte activation markers in idiopathic myositis: changes with disease activity and differences among clinical and autoantibody subgroups

(2010) F. W. MILLER et al.   CLINICAL AND EXPERIMENTAL IMMUNOLOGY

Etiology and risk of lactescent plasma and severe hypertriglyceridemia

(2010) Karine Tremblay et al.   Journal of Clinical Lipidology

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

(2009) F. Mingozzi et al.   BLOOD

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Gene Therapy Using Adeno-Associated Virus Vectors

(2008) S. Daya et al.   CLINICAL MICROBIOLOGY REVIEWS

Enhanced susceptibility to pancreatitis in severe hypertriglyceridaemic lipoprotein lipase-deficient mice and agonist-like function of pancreatic lipase in pancreatic cells

(2008) Y Wang et al.   GUT

Infectious Molecular Clones of Adeno-Associated Virus Isolated Directly from Human Tissues

(2008) B. C. Schnepp et al.   JOURNAL OF VIROLOGY