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

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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Bibliographic Details
Main Authors: Young Jae Moon, Ravi Hindupur, Iteoluwakishi H. Gamu, Nikki M. McCormack, Fatima Shaikh, James S. Novak, Jyoti K. Jaiswal
Format: Article
Language:English
Published: Elsevier 2025-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
Duchenne muscular dystrophy, DMD
fibrosis
myogenesis
inflammation
fibroadipogenic
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125002197
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http://www.sciencedirect.com/science/article/pii/S2162253125002197

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