Splice-Switching Antisense Oligonucleotides: 30 Years after Advocating Exon-Skipping Therapy for Duchenne Muscular Dystrophy
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".
Deadline for manuscript submissions: 20 February 2025 | Viewed by 643
Special Issue Editor
Special Issue Information
Dear Colleagues,
The year 2025 marks 30 years since we proposed a treatment for Duchenne muscular dystrophy (D MD) using antisense oligonucleotides (ASOs) to induce exon skipping, and consequently, dystrophin expression. When we proposed this treatment in 1995, we were afraid it would fizzle out, like a match lit in a cold sky. However, the development of splice-switching ASO-based therapies has spread like wildfire over the past 30 years. Today, four ASOs that induce exon skipping, as DMD therapies, offer great hope for patients worldwide. In addition, one ASO that promotes exon incorporation into mRNA has produced a treatment for spinal muscular atrophy. Recently, an ASO that treats only one patient has presented us with the ultimate personalized medicine. In addition, ASOs that carry out splice switching have been applied to redirect gene function for the effective treatment of diseases. At the same time, studies on the chemical modification or delivery of nucleic acids for improving the efficacy of ASOs are also making great progress. This Special Issue highlights studies on splice-switching ASOs in these various areas, which we hope will serve as a cornerstone for the future of ASO drugs. We welcome submissions from the many researchers involved in ASO studies.
Prof. Dr. Masafumi Matsuo
Guest Editor
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Keywords
- antisense oligonucleotides
- modified nucleic acid
- splicing
- splicing switch
- exon skipping
- pseudoexon
- N-of-1
- precision medicine
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