Muscular Dystrophy: From Mechanisms to Therapeutic Approaches
A special issue of Biomedicines (ISSN 2227-9059). This special issue belongs to the section "Molecular Genetics and Genetic Diseases".
Deadline for manuscript submissions: 30 April 2026 | Viewed by 43
Special Issue Editors
Interests: muscular dystrophy; pathophysiological mechanisms; muscle regeneration; molecular diagnosis; therapeutic approaches
Special Issues, Collections and Topics in MDPI journals
Interests: neuromuscular diseases; proteomics; cell death
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Muscular dystrophies represent a highly heterogeneous group of disorders characterized by progressive muscle weakness, impaired regeneration, and multisystem involvement. Despite advances in clinical diagnosis and supportive care, there is still no definitive cure for most of these conditions, highlighting the urgent need for a deeper understanding of disease mechanisms and the development of effective therapies.
In recent years, significant progress has been achieved through the integration of molecular genetics, omics technologies, and disease modeling. Novel insights into gene mutations, RNA processing, mitochondrial dysfunction, and dysregulated signaling pathways have provided a more comprehensive picture of disease pathogenesis. At the same time, therapeutic strategies are rapidly evolving, ranging from gene replacement and genome editing to antisense oligonucleotides, cell-based therapies, and the pharmacological modulation of key pathways.
This Special Issue aims to bring together cutting-edge research and reviews focusing on the molecular and cellular mechanisms underlying muscular dystrophies, as well as innovative preclinical and clinical therapeutic approaches. Contributions spanning basic science, translational research, and clinical studies are welcome, with the aim of fostering multidisciplinary dialogue and advancing personalized medicine for these challenging diseases.
Dr. Gisela Gaina
Dr. Marco A.F. Fernandes
Guest Editors
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Keywords
- muscular dystrophy
- genetic diseases
- muscle regeneration
- therapeutic approaches
- molecular diagnosis
- in vitro and in vivo models
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