Lipid nanoparticle-targeted mRNA formulation as a treatment for ornithine-transcarbamylase deficiency model mice

Ornithine transcarbamylase (OTC) plays a significant role in the urea cycle, a metabolic pathway functioning in the liver to detoxify ammonia. OTC deficiency (OTCD) is the most prevalent urea cycle disorder. Here, we show that intravenously delivered human OTC (hOTC) mRNA by lipid nanoparticles (LNP...

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Bibliographic Details

Published in:	Molecular Therapy: Nucleic Acids
Main Authors:	Kazuto Yamazaki, Kenji Kubara, Satoko Ishii, Keita Kondo, Yuta Suzuki, Takayuki Miyazaki, Kaoru Mitsuhashi, Masashi Ito, Kappei Tsukahara
Format:	Article
Language:	English
Published:	Elsevier 2023-09-01
Subjects:	MT: delivery strategies: ornithine transcarbamylase messenger RNA therapy lipid nanoparticles homotrimer conformation cryo-electron microscopy mitochondria localization
Online Access:	http://www.sciencedirect.com/science/article/pii/S2162253123001737