Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemiaResearch in context

Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemiaResearch in context

Background: Isolated methylmalonic acidemia/aciduria (MMA) is an ultra-rare, serious, inherited metabolic disorder with significant morbidity and mortality. Exogenously delivered mRNA encoding human methylmalonyl-CoA mutase (hMUT), the enzyme most frequently mutated in MMA, is a potential therapy to...

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Bibliographic Details
Main Authors: Ding An, Andrea Frassetto, Eric Jacquinet, Marianne Eybye, Joseph Milano, Christine DeAntonis, Vi Nguyen, Rodrigo Laureano, Jaclyn Milton, Staci Sabnis, Christine M. Lukacs, Lin T. Guey
Format: Article
Language:English
Published: Elsevier 2019-07-01
Series:EBioMedicine
Online Access:http://www.sciencedirect.com/science/article/pii/S2352396419304384

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http://www.sciencedirect.com/science/article/pii/S2352396419304384

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