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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Detalhes bibliográficos

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
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Wiley 2022-06-01
Colecção:	Clinical and Translational Discovery
Assuntos:	liver biopsy LUNAR‐OTC ornithine transcarbamylase (OTC) OTC activity assay ornithine transcarbamylase deficiency (OTCD) plasma
Acesso em linha:	https://doi.org/10.1002/ctd2.33
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