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Article

Delivering the Message: Translating mRNA Therapy for Liver Inherited Metabolic Diseases

18 Aug 2025

Abstract excerpt

mRNA encapsulated in lipid nanoparticles (LNPs) provides a dual revolution in the field of gene therapy. mRNA brings fleeting efficacy and the possibility to adjust the therapy to clinical needs. LNP, as a non-viral vehicle with flexible organ-targeting, overcomes most immune complications of viral gene therapy. mRNA-LNP has rapidly progressed from preventive medicine and vaccine applications to therapeutic use,...

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PMID: 40826824
DOI: 10.1002/jimd.70078

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