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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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Principais autores: 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
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2026-03-01
Colecção:Molecular Therapy: Nucleic Acids
Assuntos:
MT: Oligonucleotides: Therapies and Applications
Duchenne Muscular Dystrophy
exon skipping therapy
PPMO
breathing
respiratory dysfunction
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S2162253125003646
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