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

• Main Authors: Kazuto Yamazaki (Author), Kenji Kubara (Author), Satoko Ishii (Author), Keita Kondo (Author), Yuta Suzuki (Author), Takayuki Miyazaki (Author), Kaoru Mitsuhashi (Author), Masashi Ito (Author), Kappei Tsukahara (Author)
• Format: Book
• Published: Elsevier, 2023-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...