Improving angiogenesis ameliorates the efficacy of ASO-based exon skipping for the treatment of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a severe X-linked disease caused by pathogenic variants in the DMD gene, resulting in the absence of functional dystrophin. Antisense oligonucleotide (ASO)-based therapies aim to restore the open reading frame and produce a truncated but functional dystrophin pro...

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Principais autores:	Mathilde Blitek, Cécile Gastaldi, Mathilde Doisy, Olivier Le Coz, Marion David, Xaysongkhame Phongsavanh, Sameh Ben Aicha, Luis Garcia, Alessio Rotini, Gilles Pagès, Aurélie Goyenvalle
Formato:	Artigo
Idioma:	Inglês
Publicado:	Elsevier 2026-03-01
Series:	Molecular Therapy: Nucleic Acids
Assuntos:	MT: Oligonucleotides: Therapies and Applications Duchenne muscular dystrophy exon skipping splice switching combined therapy microvasculature
Acceso en liña:	http://www.sciencedirect.com/science/article/pii/S2162253126000053
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