Antisense and gene therapy trials in Duchenne muscular dystrophy

Mariacristina Scoto; Francesco Muntoni

doi:10.1016/j.medj.2025.100938

Antisense and gene therapy trials in Duchenne muscular dystrophy

Med. 2025 Dec 12;6(12):100938. doi: 10.1016/j.medj.2025.100938.

Authors

Mariacristina Scoto¹, Francesco Muntoni²

Affiliations

¹ Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health and Institute of Neurology, University College London, London, UK.
² Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health and Institute of Neurology, University College London, London, UK. Electronic address: f.muntoni@ucl.ac.uk.

PMID: 41389707
DOI: 10.1016/j.medj.2025.100938

Abstract

Duchenne muscular dystrophy (DMD) is severe and the most common form of muscular dystrophy in childhood. There is no cure for this condition, but several approved therapeutic approaches aim to delay disease progression. Multiple ongoing clinical trials attempt to address the root cause of the condition: exon skipping antisense oligonucleotides (ASOs-targeting specific mutations) and AAV-mediated gene replacement therapy.

Publication types

Review

MeSH terms

Clinical Trials as Topic
Dependovirus / genetics
Dystrophin / genetics
Exons
Genetic Therapy* / methods
Humans
Muscular Dystrophy, Duchenne* / genetics
Muscular Dystrophy, Duchenne* / therapy
Oligonucleotides, Antisense* / therapeutic use

Substances

Oligonucleotides, Antisense
Dystrophin