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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Autores principales:	Young Jae Moon, Ravi Hindupur, Iteoluwakishi H. Gamu, Nikki M. McCormack, Fatima Shaikh, James S. Novak, Jyoti K. Jaiswal
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	Elsevier 2025-09-01
Colección:	Molecular Therapy: Nucleic Acids
Materias:	MT: Oligonucleotides: Therapies and Applications Duchenne muscular dystrophy, DMD fibrosis myogenesis inflammation fibroadipogenic
Acceso en línea:	http://www.sciencedirect.com/science/article/pii/S2162253125002197
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