PPMO-based exon skipping therapy improves respiratory function in the mdx mouse model of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a severe X-linked recessive disorder caused by a deficiency of dystrophin, leading to progressive muscle degeneration and eventually cardiorespiratory failure. Exon-skipping therapies using cell-penetrating peptide-conjugated phosphorodiamidate morpholino oligome...

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Autori principali:	Debolina D. Biswas, Maran Y. Hernandez Rodriguez, Lea El Haddad, Gwendolyn Hoffmann, Elena Copson, Sloane Mayman, Julia Jose, Sydney Lang, Olivia Chen, Andrea Dela Pena, Emerson Levin, Mariam Abdelbarr, Aoife Slyne, Mai K. ElMallah
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2026-03-01
Serie:	Molecular Therapy: Nucleic Acids
Soggetti:	MT: Oligonucleotides: Therapies and Applications Duchenne Muscular Dystrophy exon skipping therapy PPMO breathing respiratory dysfunction
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2162253125003646
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