Gene Therapy for Rare Disorders
A special issue of Genes (ISSN 2073-4425). This special issue belongs to the section "Human Genomics and Genetic Diseases".
Deadline for manuscript submissions: 25 May 2025 | Viewed by 189
Special Issue Editor
Special Issue Information
Dear Colleagues,
Genetic disorders present mild to fatal consequences to individuals, with limitations imposed beyond the patient to family members and with tremendously high societal costs. Gene therapy represents one of the most promising developments in modern medicine. The rise in new technologies in gene-based medicines is rapidly transforming the medical field from one of diagnosis and supportive care to one of numerous clinical trials and an increasing number of approved treatments.
Gene therapy encompasses all work aimed at editing DNA or altering the transcript or epigenetic profile to incur a therapeutic benefit. These include studies relating to gene augmentation therapy, gene editing, or gene silencing.
For almost five decades, researchers have sought to manipulate the natural infection process of viruses to incorporate exogenous, corrective DNA into host cells. Along the way, this field has expanded to include the direct editing of DNA, RNA silencing, and methylation editing, resulting in an altered epigenetic profile.
As of 2023, approximately 3900 gene therapy clinical trials have been completed. Each year, new therapies are brought onto the market with the potential to save the lives of numerous patients suffering from rare diseases.
We solicit manuscripts relating to gene augmentation, gene editing, gene silencing, and epigenetic manipulation, where the intent is to develop therapy for rare diseases. We are also soliciting manuscripts that seek to enhance the safety profile of gene therapy interventions.
Dr. Ryan Butler
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.
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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.
Keywords
- gene therapy
- rare diseases
- viral vectors
- RNA silencing
- gene editing
- methylation editing
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