Article
Gene therapy for genetic lipoprotein lipase deficiency: from promise to practice.
The Netherlands journal of medicine - 1 Jan 2005
Nierman M C, Rip J, Twisk J, Meulenberg J J M, Kastelein J J P, Stroes E S G, Kuivenhoven J A
Abstract excerpt
Lipoprotein lipase (LPL) deficiency is a rare, hereditary disorder of lipoprotein metabolism characterised by severely increased triglyceride levels, and associated with an increased risk for pancreatitis. Since no adequate treatment modality is available for this disorder, we set out to develop an LPL gene therapy protocol. This paper focuses on the clinical presentation of LPL deficiency, summarises the...
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