Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia.

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

EBioMedicine（2019）

引用 42|浏览12

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摘要

These data demonstrate durable functional benefit of hMUT mRNA and support development of this new class of therapy for a devastating, pediatric disorder. FUND: This work was funded by Moderna, Inc.

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关键词

Enzyme replacement,Liver targeted therapy,Methylmalonyl-CoA mutase,Organic acidemia,Systemic mRNA therapy

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