Special Issue "Mechanisms and Novel Therapeutic Approaches for Muscle Disease"
Deadline for manuscript submissions: 31 December 2023 | Viewed by 12052
Interests: neuromuscular disorders; pathophysiological mechanisms; RNA therapeutics; viral vector-based gene therapy
2. Department of Biological Sciences, School of Life Sciences and the Environment, Royal Holloway University of London, Egham TW20 0EX, UK
Interests: rare disease; neuromuscular disorders; cancers; pathophysiological mechanisms; targeted therapeutics; antisense oligonucleotides; genome editing; gene addition
Muscular dystrophy is a group of inherited rare diseases that causes progressive weakness and the degeneration of skeletal and cardiac muscles, with or without damaging the nerve supplied to the muscles. The diseases often affect a particular group of muscles before spreading more widely to other muscle groups, increasing the level of disability and possible deformity or even becoming life-threatening when cardiac or/and respiratory muscles are severely impaired. The global prevalence of muscular dystrophy is estimated at 3.6 per 100,000 people.
Despite being a genetic condition, it usually takes five years to properly diagnose a muscular disorder. However, disease management mostly involves symptomatic and palliative care as there is a lack of curative treatments available. Understanding the pathophysiological mechanisms underlying muscular diseases is, therefore, crucial. This is the most effective way to provide appropriate support for the development of therapeutic strategies for addressing the root cause of the pathology.
This Special Issue on “Mechanisms and Novel Therapeutic Approaches for Muscle Disease” therefore welcomes basic to translational research studies that aim to provide up-to-date insights into pathophysiological mechanisms, innovative methodologies, cellular and animal models, and novel therapeutic strategies that have a great potential for the development of a cure or modifying treatments for muscular dystrophies.
Dr. Ngoc B. Lu-Nguyen
Prof. Dr. Linda Popplewell
Manuscript Submission Information
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Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). 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.
- muscular dystrophy
- pathophysiological mechanisms
- innovative methodologies
- cellular and animal models
- therapeutic strategies