Gene and Cell Therapy: New Findings from Medical Research and Treatment

Dear Colleagues,

Gene and cell therapies represent a rapidly evolving area of biomedical research with the potential to fundamentally transform the prevention, diagnosis, and treatment of a wide range of human diseases. These innovative approaches are being actively explored for single-gene and polygenic disorders, cancer, cardiovascular and vascular diseases, neurodegenerative disorders, inflammatory and autoimmune conditions, infectious diseases, acquired pathologies, and more. By enabling highly targeted and patient-specific interventions, gene and cell therapies constitute a cornerstone of precision and personalized medicine.

This Special Issue aims to highlight cutting-edge advances in gene and cell therapy across a broad disease spectrum, with particular emphasis on the translational continuum from basic research to clinical application. We seek to showcase studies that elucidate disease mechanisms at the molecular and cellular levels and translate these insights into innovative diagnostic tools and therapeutic strategies. Contributions that integrate knowledge of disease etiology, pathogenesis, and treatment modalities—bridging laboratory discoveries with preclinical development and clinical evaluation—are especially encouraged.

We welcome original research articles, reviews, and clinical or translational studies that explore the development, optimization, and application of gene- and cell-based therapies. Submissions may address fundamental biological mechanisms, novel delivery systems, therapeutic targets, predictive and prognostic biomarkers, or early- and late-phase clinical studies. Collectively, these contributions should provide a comprehensive view of how gene and cell therapies can be effectively translated from bench to bedside to improve patient outcomes across diverse medical fields.

The scope of this Special Issue includes, but is not limited to, the following topics:

• Gene and cell therapy for genetic, metabolic, cardiovascular, neurological, inflammatory, infectious, and neoplastic diseases
• Immunotherapy and immune-cell-based therapies
• Adoptive cell therapy (e.g., CAR-T-, CAR-NK-, TCR-based approaches)
• Stem cell therapy and regenerative medicine
• Tissue engineering and organ repair
• Epigenetics and stem cell biology
• Genome editing technologies
• RNA-based therapeutics
• Viral and non-viral (including bacterial) vectors
• Oncolytic viruses and therapeutic vaccines
• Disease-associated biomarkers and patient stratification
• Translational and clinical studies of gene and cell therapies

By bringing together basic scientists, translational researchers, and clinicians, this Special Issue aims to provide a multidisciplinary platform that advances our understanding of gene and cell therapy and facilitates their successful clinical implementation across a wide range of diseases.

Dr. Lorella Tripodi
Dr. Antonio Di Stasi
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 250 words) can be sent to the Editorial Office for assessment.

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-anonymized 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 Translational Medicine is an international peer-reviewed open access quarterly 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 1200 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.