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...
Main Authors: | , , , , , , , , , , , |
---|---|
Format: | Article |
Language: | English |
Published: |
Elsevier
2019-07-01
|
Series: | EBioMedicine |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2352396419304384 |