Genetic Diagnosis and Treatment of Duchenne Muscular Dystrophy
A special issue of Genes (ISSN 2073-4425). This special issue belongs to the section "Molecular Genetics and Genomics".
Deadline for manuscript submissions: 20 December 2025 | Viewed by 77
Special Issue Editor
Interests: viral and non-viral gene therapies; neuromuscular and cardiovascular diseases
Special Issue Information
Dear Colleagues,
We are delighted to invite submissions for the Special Issue, “Genetic Diagnosis and Treatment of Duchenne Muscular Dystrophy”.
Duchenne muscular dystrophy (DMD) is one of the most common forms of muscular dystrophy, caused by mutations in the DMD gene encoding dystrophin protein, the deficiency or absence of which leads to progressive muscle weakness, cardiac and respiratory failure, and premature death. The DMD gene is the largest gene in the human genome with a highly diverse mutational profile that includes single and multiple exon duplications and deletions, as well as single-point and frameshift mutations that disrupt the dystrophin open reading frame. Over the past few decades, many therapeutic strategies have been developed and tested, some of which have received regulatory approval, and we expect that more will follow.
This Special Issue seeks to highlight advances in basic, translational, and clinical research to understand DMD at the molecular and structural levels. We welcome original research articles and reviews that highlight the development of meaningful therapies for DMD using cutting-edge technologies, biomarkers, and methods that standardize the functional outcome measures needed to assess therapeutic efficacy.
Original research articles and reviews will be considered for publication.
Dr. Liubov V. Gushchina
Guest Editor
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. Genes is an international peer-reviewed open access monthly journal published by MDPI.
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.
Keywords
- Duchenne muscular dystrophy
- viral vectors
- non-viral therapy
- gene editing
- common and rare mutations
- biomarkers
- methods of diagnosis
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