Work

Myomerger-derived peptide enhances skeletal muscle tropism and reduces liver transduction of lipid nanoparticles for gene delivery

Year: 2025

Type: article

Abstract: , MyomP1-LNPs significantly enhanced muscle transduction when delivering DNA cargo, strongly reduced liver accumulation following intramuscular and intravenous mRNA delivery, and attenuated local immune activation. This work demonstrates a ligand-guided strategy to overcome organ-specific barriers in nonviral gene transfer, with improved safety and specificity. It suggests that MyomP1-engineered LNPs hold strong potential to improve therapeutic outcomes for patients with rare muscle diseases, offering a promising alternative to traditional viral gene therapy platforms.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1016/j.omtn.2025.102785

Authors: Jacqueline Ji, Eva Lipkow, Nicolas Anton, Corinne Crucifix, Pascal Eberling, Jocelyn Laporte