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Article

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

28 Aug 2013

Abstract excerpt

BACKGROUND: Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated by RNA interference (RNAi) could reduce the production of transthyretin. METHODS: We identified a potent antitransthyretin small interfering RNA, which was encapsulated in two distinct first- and second-generation formulations of lipid...

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PMID: 23984729
DOI: 10.1056/nejmoa1208760

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