Novel Therapies and Biomarkers for Rare Diseases
A special issue of Cells (ISSN 2073-4409).
Deadline for manuscript submissions: 15 November 2025 | Viewed by 27
Special Issue Editor
Special Issue Information
Dear Colleagues,
This Special Issue invites high-quality original research and review articles focused on advancing cellular and molecular understanding of rare diseases through cutting-edge therapeutic and diagnostic innovations. We encourage submissions that explore innovative therapeutic approaches including small molecules, biologics, gene therapies, cell-based interventions, and novel biomarker discovery using omics approaches.
Contributions that investigate fundamental cellular processes, such as signaling, metabolism, autophagy, apoptosis, immunology, stem cell biology, and neurodegeneration, in the context of rare diseases and shed light on therapeutic strategies are highly welcome. Studies using genomics, transcriptomics, proteomics, and metabolomics to identify novel disease biomarkers and therapeutic targets are especially encouraged.
Rare diseases, though individually uncommon, collectively affect millions worldwide. However, therapies and diagnostics remain limited. By focusing on mechanisms such as genetic mutations, cellular dysfunction, and systemic effects, this Special Issue will bridge basic and translational research by highlighting new directions in rare disease therapy and diagnostics, fostering collaborations across disciplines.
We welcome submissions covering preclinical models, clinical applications (i.e., use of clinical samples for biomarker discovery), cell-based therapies, and innovative drug development platforms. By gathering cutting-edge research, this Special Issue will contribute to the broader mission of Cells in advancing cellular and molecular medicine.
Dr. Reena Kartha
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. Cells is an international peer-reviewed open access semimonthly 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 2700 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
- rare diseases
- translational research
- novel therapies
- biomarker discovery
- precision medicine
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