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Translational Pharmacokinetic/Pharmacodynamic Model for mRNA-3927, an Investigational Therapeutic for the Treatment of Propionic Acidemia

Propionic acidemia (PA) is an ultrarare disorder caused by deficiency of the mitochondrial enzyme, propionyl-CoA carboxylase (PCC), composed of PCCA and PCCB subunits. An enzyme replacement therapy is being developed using dual messenger RNA (mRNA) therapy composed of lipid nanoparticles (LNPs) enca...

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Detalles Bibliográficos
Autores principales:	Attarwala, Husain, Lumley, Matthew, Liang, Min, Ivaturi, Vijay, Senn, Joe
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2023
Materias:	Original Papers
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10066765/ https://www.ncbi.nlm.nih.gov/pubmed/36577040 http://dx.doi.org/10.1089/nat.2022.0036

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