Neuromuscular Disorders: Current Gene and Cell Therapeutic Approaches
A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Biopharmaceuticals".
Deadline for manuscript submissions: 15 September 2024 | Viewed by 12937
Special Issue Editors
Interests: RNA biology; regulatory RNA molecules; extracellular vesicles; identification of genetic defects in inherited diseases
Special Issues, Collections and Topics in MDPI journals
Interests: muscular dystrophies; gene therapy; biomarkers; miRNAs
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Neuromuscular disorders are a heterogenic group of diseases affecting the muscular system or the peripheral nervous system. Some of these conditions are also associated with the central nervous system, such as amyotrophic lateral sclerosis (ALS). Neuromuscular disorders are predominantly characterized by progressive muscle weakness. Currently, no therapeutic treatments have been identified for any of these disorders. The high heterogeneity with respect to clinical severity, the cause of the disease, and the primary cell type that is affected (e.g., motor neurons, skeletal muscle, and Schwann cells) are considered the main challenges for the development of a successful therapeutic strategy for this group of disorders. However, in recent years, research into therapy development for neuromuscular disorders has made remarkable progress. Innovative therapeutic approaches have been applied to specific neuromuscular diseases with encouraging results.
This Special Issue will cover the recent progress in developing therapeutic and diagnostic strategies for neuromuscular disorders through a series of original research articles and reviews from experts in the field. Therapeutic approaches involving the use of nucleic acid oligonucleotides such as antisense oligonucleotides and miRNAs, biopharmaceutical compounds, genome editing, and different delivery tools including viral vectors and nanoparticles will be highlighted in this Special Issue.
Prof. Dr. Leonidas A. Phylactou
Dr. Andrie Koutsoulidou
Guest Editors
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Keywords
- Muscular dystrophies
- Motor neuron diseases
- Myopathies
- Cell-based therapy
- Gene therapy
- Antisense oligonucleotides
- miRNAs
- Pharmacological strategies
- Stem cells
- Viral vectors
- Nanoparticles