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 253
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
Manuscript Submission Information
Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.
Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.
Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.
Keywords
- antisense oligonucleotides
- modified nucleic acid
- splicing
- splicing switch
- exon skipping
- pseudoexon
- N-of-1
- precision medicine
Benefits of Publishing in a Special Issue
- Ease of navigation: Grouping papers by topic helps scholars navigate broad scope journals more efficiently.
- Greater discoverability: Special Issues support the reach and impact of scientific research. Articles in Special Issues are more discoverable and cited more frequently.
- Expansion of research network: Special Issues facilitate connections among authors, fostering scientific collaborations.
- External promotion: Articles in Special Issues are often promoted through the journal's social media, increasing their visibility.
- e-Book format: Special Issues with more than 10 articles can be published as dedicated e-books, ensuring wide and rapid dissemination.
Further information on MDPI's Special Issue polices can be found here.
