Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia

Propionic acidemia/aciduria (PA) is an ultra-rare, life-threatening, inherited metabolic disorder caused by deficiency of the mitochondrial enzyme, propionyl-CoA carboxylase (PCC) composed of six alpha (PCCA) and six beta (PCCB) subunits. We herein report an enzyme replacement approach to treat PA u...

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Detalles Bibliográficos
Autores principales: Jiang, Lei, Park, Ji-Sun, Yin, Ling, Laureano, Rodrigo, Jacquinet, Eric, Yang, Jinsong, Liang, Shi, Frassetto, Andrea, Zhuo, Jenny, Yan, Xinhua, Zhu, Xuling, Fortucci, Steven, Hoar, Kara, Mihai, Cosmin, Tunkey, Christopher, Presnyak, Vlad, Benenato, Kerry E., Lukacs, Christine M., Martini, Paolo G. V., Guey, Lin T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7578066/
https://www.ncbi.nlm.nih.gov/pubmed/33087718
http://dx.doi.org/10.1038/s41467-020-19156-3

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