{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Du X"],"funding":["Japan Science and Technology Agency","Japan Agency for Medical Research and Development (AMED)"],"pagination":["e2409065"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12021044"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["12(16)"],"pubmed_abstract":["Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, leading to the absence of dystrophin and progressive muscle degeneration. Current therapeutic strategies, such as exon-skipping and gene therapy, face limitations including truncated dystrophin production and safety concerns. To address these issues, a novel mRNA-based therapy is explored using polyplex nanomicelles to deliver mRNA encoding peroxisome proliferator-activated receptor gamma coactivator 1 alpha isoform 4 (PGC-1α4) via hydrodynamic limb vein (HLV) administration. Using an in vivo muscle torque measurement technique, it is observed that nanomicelle-delivered Pgc-1α4 mRNA significantly improved muscle damage resistance and mitochondrial activity in mdx mice. Specifically, HLV administration of Pgc-1α4 mRNA"],"journal":["Advanced science (Weinheim, Baden-Wurttemberg, Germany)"],"pubmed_title":["Polyplex Nanomicelle-Mediated Pgc-1α4 mRNA Delivery Via Hydrodynamic Limb Vein Injection Enhances Damage Resistance in Duchenne Muscular Dystrophy Mice."],"pmcid":["PMC12021044"],"funding_grant_id":["JPMJPF2202","JP223fa627002","23fk0310515s0502","23ek0109659h0001","23ak0101173s0203"],"pubmed_authors":["Minegishi K","Sin Y","Itaka K","Nakanishi H","Yamada T","Motohashi N","Du X","Aoki Y"],"additional_accession":[]},"is_claimable":false,"name":"Polyplex Nanomicelle-Mediated Pgc-1α4 mRNA Delivery Via Hydrodynamic Limb Vein Injection Enhances Damage Resistance in Duchenne Muscular Dystrophy Mice.","description":"Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, leading to the absence of dystrophin and progressive muscle degeneration. Current therapeutic strategies, such as exon-skipping and gene therapy, face limitations including truncated dystrophin production and safety concerns. To address these issues, a novel mRNA-based therapy is explored using polyplex nanomicelles to deliver mRNA encoding peroxisome proliferator-activated receptor gamma coactivator 1 alpha isoform 4 (PGC-1α4) via hydrodynamic limb vein (HLV) administration. Using an in vivo muscle torque measurement technique, it is observed that nanomicelle-delivered Pgc-1α4 mRNA significantly improved muscle damage resistance and mitochondrial activity in mdx mice. Specifically, HLV administration of Pgc-1α4 mRNA","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Apr","modification":"2026-07-15T08:50:46.635Z","creation":"2025-07-11T03:05:54.944Z"},"accession":"S-EPMC12021044","cross_references":{"pubmed":["40051178"],"doi":["10.1002/advs.202409065"]}}