Unlocking the Future of Muscle Disease Treatment: Advancements in Gene Therapy, Antisense Therapeutics and Cutting-Edge Pharmacological Strategies

Dear Colleagues,

Muscle disorders, including muscular dystrophy and various neuromuscular diseases, are remarkably heterogenic in terms of genetic defects, pathophysiology, and the age of onset. Unfortunately, effective treatments for these disorders have been elusive, principally due to their rarity and the necessity of often mutation-specific approaches and tailored therapies. However, in recent years, we have witnessed the emergence of novel therapies, such as gene therapy, antisense therapeutics, and pharmacological approaches. These breakthroughs have shown promise for the treatment of diseases like Duchenne muscular dystrophy and spinal muscular atrophy and ongoing advancements in the development of treatments for myotubular myopathy or limb girdle muscular dystrophy. These encouraging advancements demonstrate the feasibility of developing treatments for severe and complex muscle diseases using new tools and models and via collaboration between preclinical research laboratories and industries.

In light of these remarkable advancements, we invite scholars to contribute to our Special Issue on “Unlocking the Future of Muscle Disease Treatment: Advancements in Gene Therapy, Antisense Therapeutics and Cutting-Edge Pharmacological Strategies”. We encourage the submission of manuscripts describing basic and translational research studies. We aim to provide the latest insights into the development of innovative methodologies, including in silico and bioinformatics approaches, as well as advancements in in vitro and in vivo models. Ultimately, this Special Issue seeks to shed light on the progress and potential of innovative therapeutic strategies for the treatment of muscular diseases.

Dr. Ngoc B. Lu-Nguyen
Dr. Alberto Malerba
Guest Editors

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.

• muscle disease
• antisense therapeutics
• gene therapy
• pharmacological approach
• bioinformatic tools
• cellular models
• animal models