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

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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Bibliographic Details
Main Authors: 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: Article
Language:English
Published: Wiley 2022-06-01
Series:Clinical and Translational Discovery
Subjects:
liver biopsy
LUNAR‐OTC
ornithine transcarbamylase (OTC)
OTC activity assay
ornithine transcarbamylase deficiency (OTCD)
plasma
Online Access:https://doi.org/10.1002/ctd2.33

Internet

https://doi.org/10.1002/ctd2.33

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