A combinatorial oligonucleotide therapy to improve dystrophin restoration and dystrophin-deficient muscle health

Despite the proven safety of dystrophin-targeting phosphorodiamidate morpholino oligomer (PMO) therapy, poor delivery of the PMOs limit the efficacy of this dystrophin restoring gene therapy for Duchenne muscular dystrophy (DMD). Limited myogenesis and excessive fibrosis in DMD are pathological feat...

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Auteurs principaux:	Young Jae Moon, Ravi Hindupur, Iteoluwakishi H. Gamu, Nikki M. McCormack, Fatima Shaikh, James S. Novak, Jyoti K. Jaiswal
Format:	Artigo
Langue:	Inglês
Publié:	Elsevier 2025-09-01
Collection:	Molecular Therapy: Nucleic Acids
Sujets:	MT: Oligonucleotides: Therapies and Applications Duchenne muscular dystrophy, DMD fibrosis myogenesis inflammation fibroadipogenic
Accès en ligne:	http://www.sciencedirect.com/science/article/pii/S2162253125002197
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