Polyplex Nanomicelle‐Mediated Pgc‐1α4 mRNA Delivery Via Hydrodynamic Limb Vein Injection Enhances Damage Resistance in Duchenne Muscular Dystrophy Mice

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 c...

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Autors principals:	Xuan Du, Hideyuki Nakanishi, Takashi Yamada, Yooksil Sin, Katsura Minegishi, Norio Motohashi, Yoshitsugu Aoki, Keiji Itaka
Format:	Artigo
Idioma:	Inglês
Publicat:	Wiley 2025-04-01
Col·lecció:	Advanced Science
Matèries:	duchenne muscular dystrophy (DMD) lipid nanoparticle (LNP) mRNA therapeutics peroxisome proliferator‐activated receptor gamma coactivator (PGC‐1α) polyplex nanomicelle
Accés en línia:	https://doi.org/10.1002/advs.202409065
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