Related Experiment Video
Updated: Jan 8, 2026

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
Published on: May 7, 2020
Antisense and gene therapy trials in Duchenne muscular dystrophy
Mariacristina Scoto1, Francesco Muntoni1
1Dubowitz 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.
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.
Related Concept Videos
Gene Therapy
Satellite Stem Cells and Muscular Dystrophy

