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Case studies of pipeline candidates in oncology, cardiology, and rare diseases In propionic acidemia (PA) mouse models, systemic LNP delivery of dual mRNA-3927 encoding both PCC subunits restored hepatic enzyme activity and significantly reduced levels of disease biomarkers, with consistent efficacy over 36 months post-dose and robust pharmacokinetics across mice, rats, and nonhuman primates supporting rapid protein expression within 6 h and informing first-in-human dosing [201]

ACSL4, an enzyme that converts fatty acid to fatty acyl-CoA esters, is known to dictate ferroptosis sensitivity by shaping cellular lipid composition (27) and contributes to intestinal tissue injury (68)

The presence of murine macrophages in this area further supports the formation of this immune hub (Fig

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[DOI] [PubMed] [Google Scholar] 73.Qian X., Lu Z., Tan M., Liu H., Lu D