Improving angiogenesis ameliorates the efficacy of ASO-based exon-skipping for the treatment of Duchenne muscular dystrophy
Mathilde Blitek, Cécile Gastaldi, Mathilde Doisy, Olivier Le Coz, Marion David, Xaysongkhame Phongsavanh, Sameh Ben Aicha, Luis Garcı́a, Alessio Rotini, Gilles Pagès, Aurélie Goyenvalle
This study shows that improving angiogenesis enhances ASO-based exon-skipping therapy in DMD by increasing ASO delivery and dystrophin expression in mdx mice.
Molecular Therapy — Nucleic Acids · Jan 13, 2026Peer-reviewedMolecular Biology