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

Mathilde Blitek; Cécile Gastaldi; Mathilde Doisy dkk.

Molecular Therapy: Nucleic Acids, Vol. 37 No. 1 · 2026 · Inggris · DOAJ Scopus

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…