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Article

Gene therapy for lipoprotein lipase deficiency

13 Jun 2012

Abstract excerpt

PURPOSE OF REVIEW: The present review summarizes the clinical development of adeno-associated viral vector (AAV)1-lipoprotein lipase (LPL)S447X gene therapy (alipogene tiparvovec) for lipoprotein lipase deficiency. Lipoprotein lipase deficiency is a rare inherited disease characterized by severe hypertriglyceridaemia, chylomicronaemia and risk of recurrent pancreatitis or other complications. AAV1-LPLS447X gene...

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PMID: 22691709
DOI: 10.1097/mol.0b013e3283555a7e

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