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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書誌詳細

出版年:	Molecular Therapy: Nucleic Acids
主要な著者:	Young Jae Moon, Ravi Hindupur, Iteoluwakishi H. Gamu, Nikki M. McCormack, Fatima Shaikh, James S. Novak, Jyoti K. Jaiswal
フォーマット:	論文
言語:	英語
出版事項:	Elsevier 2025-09-01
主題:	MT: Oligonucleotides: Therapies and Applications Duchenne muscular dystrophy, DMD fibrosis myogenesis inflammation fibroadipogenic
オンライン･アクセス:	http://www.sciencedirect.com/science/article/pii/S2162253125002197