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Article

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

Nucleic acid therapeutics - 1 Apr 2023

Attarwala Husain, Lumley Matthew, Liang Min, Ivaturi Vijay, Senn Joe

Abstract excerpt

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) encapsulating mRNAs encoding PCCA and PCCB subunits of the PCC enzyme. We herein report on development of a...

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Identifiers

PMID: 36577040
DOI: 10.1089/nat.2022.0036
PMCID: PMC10066765

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