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Restoring ornithine transcarbamylase (OTC) activity in an OTC‐deficient mouse model using LUNAR‐OTC mRNA

Abstract Ornithine transcarbamylase (OTC) catalyses the reaction from ornithine to citrulline in the urea cycle. Ornithine transcarbamylase deficiency (OTCD) results in episodes of hyperammonemia. Arcturus Therapeutics developed a lipid nanoparticle (LNP)‐encapsulated OTC‐mRNA (LUNAR‐OTC) that resul...

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Principais autores: Hailong Yu, Edward Brewer, Michael Shields, Michael Crowder, Cristiano Sacchetti, Benchawanna Soontornniyomkij, Dandan Dou, Brenda Clemente, Marciano Sablad, Padmanabh Chivukula, Steve Hughes, Scott Roberts, Kumar Rajappan, Steve Tannis, Rose Sekulovich, Suezanne Parker, Pattraranee Limphong
Format: Artigo
Sprog:Inglês
Udgivet: Wiley 2022-06-01
Serier:Clinical and Translational Discovery
Fag:
liver biopsy
LUNAR‐OTC
ornithine transcarbamylase (OTC)
OTC activity assay
ornithine transcarbamylase deficiency (OTCD)
plasma
Online adgang:https://doi.org/10.1002/ctd2.33
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