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Article

Immune Responses to AAV-Vectors, the Glybera Example from Bench to Bedside

1 Jan 2014

Abstract excerpt

Alipogene tiparvovec (Glybera(®)) is an adeno-associated virus serotype 1 (AAV1)-based gene therapy that has been developed for the treatment of patients with lipoprotein lipase (LPL) deficiency. Alipogene tiparvovec contains the human LPL naturally occurring gene variant LPL(S447X) in a non-replicating viral vector based on AAV1. Such virus-derived vectors administered to humans elicit immune responses against...

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PMID: 24624131
DOI: 10.3389/fimmu.2014.00082

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