Exploring Novel Molecular Pathways and Emerging Treatments for Myotonic Dystrophy
A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Molecular Medicine".
Deadline for manuscript submissions: 15 February 2025 | Viewed by 117
Special Issue Editor
2. INCLIVA Biomedical Research Institute, Valencia, Spain
Interests: rare diseases; drug development; personalized medicine
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
We are currently living through an exciting time in the myotonic dystrophy (DM1 and DM2) field, with the first consolidated molecule developments moving through clinical trials. However, despite significant research efforts, no disease-modifying treatments are currently available for DM patients, and approaches are still limited to a few biological targets. Recent advances in understanding the molecular mechanisms underlying these diseases have opened new avenues for therapeutic intervention, offering hope for improved patient outcomes.
This Special Issue of Biomolecules will focus on the latest research and developments in the search for treatments for DM1 and DM2. We aim to present cutting-edge molecular and biochemical insights for the development of novel therapeutic strategies. Topics will include the identification and development of novel molecular targets and mechanisms connected to DM dysregulation and the identification of unique drug candidates. Additionally, we are open to exploring how combinatorial therapies around the leveraging of complementary mechanisms of action will enhance DM patients´ treatment efficacy.
We invite contributions that delve into the molecular pathways involved in DM1 and DM2, the design of therapeutic molecules, and proof-of-concept studies demonstrating the potential of new treatments. Both research and review articles are welcome, as we seek to compile a comprehensive overview of the current state and future directions in the fight against myotonic dystrophies.
Dr. Arturo Lopez-Castel
Guest Editor
Manuscript Submission Information
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Keywords
- gene therapy
- repurposing
- therapeutic oligonucleotides
- personalized medicine
- biomarkers
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