Intramuscular Administration of AAV1-Lipoprotein Lipase(S447X) Lowers Triglycerides in Lipoprotein Lipase-Deficient Patients
| Autor: | Jan Albert Kuivenhoven, Michael R. Hayden, Jaap Twisk, Erik S.G. Stroes, Katherine A. High, Aeilko H. Zwinderman, Colin J. D. Ross, Mario Maas, C. Pieter Henny, Janneke J. Meulenberg, Eleonora Aronica, Marcel Levi, John J.P. Kastelein, Remco Franssen, Melchior C. Nierman |
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| Přispěvatelé: | ACS - Amsterdam Cardiovascular Sciences, Vascular Medicine, Other Research, Anesthesiology, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, AMS - Amsterdam Movement Sciences, Radiology and Nuclear Medicine, APH - Amsterdam Public Health, Epidemiology and Data Science, ANS - Amsterdam Neuroscience, 05 Neurology and psychiatry, Pathology, Experimental Vascular Medicine, Cardiovascular Centre (CVC), Lifestyle Medicine (LM), Vascular Ageing Programme (VAP), Center for Liver, Digestive and Metabolic Diseases (CLDM) |
| Jazyk: | angličtina |
| Rok vydání: | 2008 |
| Předmět: |
medicine.medical_specialty
Time Factors Genetic Vectors Injections Intramuscular Virus Injections Viral vector chemistry.chemical_compound Internal medicine medicine Humans Triglycerides Intramuscular Lipoprotein lipase Triglyceride business.industry Vascular disease Genetic Therapy Dependovirus medicine.disease Alipogene tiparvovec Lipoprotein Lipase Endocrinology chemistry Pancreatitis Hyperlipoproteinemia Type I Cardiology and Cardiovascular Medicine business Lipoprotein |
| Zdroj: | Arteriosclerosis, thrombosis, and vascular biology, 28(12), 2303-U264. Lippincott Williams and Wilkins Arteriosclerosis, thrombosis, and vascular biology, 28(12), 2303-4. LIPPINCOTT WILLIAMS & WILKINS |
| ISSN: | 1079-5642 |
| DOI: | 10.1161/atvbaha.108.175620 |
| Popis: | Lipoprotein lipase (LPL) deficiency is a monogenetic disorder that underlies persistently elevated triglyceride (TG) levels and consequently predisposes patients to potentially life-threatening pancreatitis. In view of the absence of adequate therapy, we developed a gene replacement strategy to lower TG levels in these patients.1 This report summarizes the data of a first clinical trial (CT-AMT-010-01) in LPL-deficient individuals after intramuscular administration of a viral vector. In a 3-month open-label study, LPLS447X-adeno-associated virus subtype 1(AAV1) vector1,2 was injected in the leg musculature of 8 LPL-deficient patients at a dose of 1×1011 (n=4) or 3×1011 (n=4) genome copies per kilogram body weight (40 and 60 … |
| Databáze: | OpenAIRE |
| Externí odkaz: |
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