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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Detalles Bibliográficos
Autores principales: Yamazaki, Kazuto, Kubara, Kenji, Ishii, Satoko, Kondo, Keita, Suzuki, Yuta, Miyazaki, Takayuki, Mitsuhashi, Kaoru, Ito, Masashi, Tsukahara, Kappei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10372164/
https://www.ncbi.nlm.nih.gov/pubmed/37520683
http://dx.doi.org/10.1016/j.omtn.2023.06.023

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