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...

Celý popis

Podrobná bibliografie

Hlavní autoři:	Young Jae Moon, Ravi Hindupur, Iteoluwakishi H. Gamu, Nikki M. McCormack, Fatima Shaikh, James S. Novak, Jyoti K. Jaiswal
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2025-09-01
Edice:	Molecular Therapy: Nucleic Acids
Témata:	MT: Oligonucleotides: Therapies and Applications Duchenne muscular dystrophy, DMD fibrosis myogenesis inflammation fibroadipogenic
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S2162253125002197
Tagy:	Přidat tag Žádné tagy, Buďte první, kdo vytvoří štítek k tomuto záznamu!