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

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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Detalles Bibliográficos
Autores principales: An, Ding, Frassetto, Andrea, Jacquinet, Eric, Eybye, Marianne, Milano, Joseph, DeAntonis, Christine, Nguyen, Vi, Laureano, Rodrigo, Milton, Jaclyn, Sabnis, Staci, Lukacs, Christine M., Guey, Lin T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
Research paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6642332/
https://www.ncbi.nlm.nih.gov/pubmed/31303505
http://dx.doi.org/10.1016/j.ebiom.2019.07.003

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