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Special Issues
Gene and Cell Therapy: New Findings from Medical Research and...
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Gene and Cell Therapy: New Findings from Medical Research and Treatment

A special issue of International Journal of Translational Medicine (ISSN 2673-8937).

Deadline for manuscript submissions: 30 June 2026 | Viewed by 6331

Special Issue Editors

Dr. Lorella Tripodi

E-Mail Website
Guest Editor
Department of Molecular Medicine and Medical Biotechnologies, University of Naples Federico II, 80131 Naples, Italy
Interests: cancer; microbiome; oncolytic adenoviruses
Dr. Antonio Di Stasi

E-Mail Website
Guest Editor
Department of Medicine, Hematology-Oncology, Stem Cell Transplantation and Cellular Therapies, The University of Alabama at Birmingham, Birmingham, AL 35294, USA
Interests: adoptive cancer immunotherapy; gene therapy; hematopoietic stem cell transplantation
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Gene and cell therapies could be employed to treat single-gene disorders, polygenic disorders, various forms of cancer, vascular diseases, neurodegenerative disorders, inflammatory conditions, and acquired diseases. This may promote the development of precision medicine and transform traditional medicine. Personalized treatment plans can be formulated by considering the optimal treatment effect according to the genes, microbiome composition, environment, and lifestyle of the patient.

This Special Issue aims to present research on personalized medicine that expands our understanding of cancer biology and enables us to identify which patient populations may benefit from treatments.

We therefore welcome the submission of articles that design targeted therapeutic solutions and propose predictive biomarkers by investigating the molecular and functional properties of different cancers.

Within the field of oncology, several approaches to the targeting of cancer are available, and each are associated with distinct mechanisms of action. Recent advancements have expanded the scope and effectiveness of these therapies, improving patient outcomes by addressing critical challenges such as precision, targeting, and delivery.

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

  • Immunotherapy;
  • Adoptive Cell Therapy;
  • Stem Cell Therapy;
  • Tumor microbiome ;
  • Tissue Engineering and Regenerative Medicine;
  • Epigenetics and Stem Cell Biology;
  • Biomarkers;
  • Viral Vector;
  • Bacterial Vector;
  • Genome Editing;
  • Oncolytic Viruses;
  • RNA Therapeutics;
  • Vaccines.

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-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 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.

Keywords

  • cell therapy
  • gene therapy
  • chimeric antigen receptor T-cell immunotherapy
  • oncolitic viruses OVs
  • cancer treatment
  • engineered mesenchymal stem cells (EMSCs)
  • RNA therapeutics
  • CRISPR-Cas9 gene editing

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Further information on MDPI's Special Issue policies can be found here.

Published Papers (3 papers)

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Research

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10 pages, 537 KB  
Article
Cardiovascular Event Surveillance Following High-Dose Intravenous Mesenchymal Stem Cell Therapy: A Single-Center Real-World Observational Study
by Takaaki Matsuoka and Nana Kobayashi
Int. J. Transl. Med. 2026, 6(2), 15; https://doi.org/10.3390/ijtm6020015 - 30 Mar 2026
Viewed by 277
Abstract
Background: The long-term cardiovascular safety of high-dose intravenous mesenchymal stem cell (MSC) therapy remains insufficiently characterized in real-world clinical settings. Methods: We conducted a single-center retrospective observational study of patients who received high-dose intravenous MSC therapy. Cardiovascular events were identified through follow-up records. [...] Read more.
Background: The long-term cardiovascular safety of high-dose intravenous mesenchymal stem cell (MSC) therapy remains insufficiently characterized in real-world clinical settings. Methods: We conducted a single-center retrospective observational study of patients who received high-dose intravenous MSC therapy. Cardiovascular events were identified through follow-up records. Observed event incidence was compared descriptively with age-adjusted population reference data. Statistical analyses were performed using two-sided Poisson methods. Results: Among treated patients, a total of four cardiovascular events were recorded during follow-up. The observed incidence did not demonstrate an excess signal compared with reference population data. No clustering of events was observed in the early post-infusion period. Sensitivity analyses yielded consistent findings. Conclusions: In this real-world cohort, high-dose intravenous MSC therapy was not associated with an apparent increase in cardiovascular event incidence. Given the observational design and limited event number, larger prospective studies are warranted to further characterize long-term cardiovascular safety. Full article
(This article belongs to the Special Issue Gene and Cell Therapy: New Findings from Medical Research and Treatment)
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11 pages, 702 KB  
Article
Intra-Articular Application of Umbilical Cord-Derived Stem Cells in Patients with Chronic Shoulder Pain
by Andrés Soto-Rodríguez, Luis Felipe Deliyore-Vega, Marcelo González-Kitzing, Paula María Muñoz-Araya, Juan Antonio Valverde-Espinoza, Victor Urzola-Herrera, Vincent Giampapa and José Rafael Rojas-Solano
Int. J. Transl. Med. 2026, 6(1), 10; https://doi.org/10.3390/ijtm6010010 - 12 Feb 2026
Viewed by 773
Abstract
Background: Chronic shoulder pain is a frequent musculoskeletal complaint that significantly affects function, productivity, and quality of life. Mesenchymal stem cell (MSC)-based therapies have emerged as a potential regenerative option due to their anti-inflammatory and tissue-repair properties. This study aims to evaluate the [...] Read more.
Background: Chronic shoulder pain is a frequent musculoskeletal complaint that significantly affects function, productivity, and quality of life. Mesenchymal stem cell (MSC)-based therapies have emerged as a potential regenerative option due to their anti-inflammatory and tissue-repair properties. This study aims to evaluate the safety and short-term effectiveness of intra-articular injections of umbilical cord-derived MSCs (UC-MSCs) in patients with chronic shoulder pain. Methods: A retrospective pragmatic observational study was conducted at the Regenerative Medicine Institute in Costa Rica. Medical records were reviewed to extract clinical, sociodemographic, and treatment-related variables. The primary outcome was functional improvement measured with the American Shoulder and Elbow Surgeons (ASES) score. Changes between baseline and the 3-month follow-up were analyzed using paired tests, effect size calculations, and regression models to explore predictors of treatment response. Results: Twenty patients met the inclusion criteria. A significant improvement in shoulder function was observed, with a mean ASES increase of 17.17 points, exceeding the minimum clinically important difference of 12. Sixty percent of patients achieved clinically meaningful improvement. Effect size estimates indicated a large magnitude of change. Regression analyses showed that baseline ASES predicted follow-up scores, while higher UC-MSC doses were associated with greater functional improvement. No adverse events were documented during the study period. Conclusions: The study shows that UC-MSC therapy is a safe, minimally invasive, and clinically beneficial option for chronic shoulder pain. These findings support the therapeutic potential of MSCs and highlight the need for larger controlled studies to validate long-term efficacy and optimize treatment protocols. Full article
(This article belongs to the Special Issue Gene and Cell Therapy: New Findings from Medical Research and Treatment)
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Review

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37 pages, 1856 KB  
Review
Current and Future Directions in Immunotherapy for Gastrointestinal Malignancies
by Catherine R. Lewis, Yazan Samhouri, Christopher Sherry, Neda Dadgar, Moses S. Raj and Patrick L. Wagner
Int. J. Transl. Med. 2025, 5(3), 33; https://doi.org/10.3390/ijtm5030033 - 31 Jul 2025
Cited by 1 | Viewed by 4532
Abstract
Gastrointestinal (GI) malignancies are diverse and particularly challenging in terms of current immunotherapy but hold great opportunity for impact given that they constitute the highest cancer incidence and mortality rates worldwide. Traditional treatment options for solid GI malignancies include surgical intervention, chemotherapy, radiation, [...] Read more.
Gastrointestinal (GI) malignancies are diverse and particularly challenging in terms of current immunotherapy but hold great opportunity for impact given that they constitute the highest cancer incidence and mortality rates worldwide. Traditional treatment options for solid GI malignancies include surgical intervention, chemotherapy, radiation, or a combination of these treatments. Emerging modalities within immunotherapy are anticipated to extend the results with conventional therapy by stimulating the patient’s own intrinsic potential for tumor-specific immunologic rejection. Combination regimens of chemotherapy and tumor-infiltrating lymphocyte (TIL) therapy in advanced colorectal cancer and pancreatic cancer, autologous monocyte therapy in advanced gastric cancer, and CAR-T therapy trained against GI-selective tumor antigens such as carcinoembryonic antigen are currently being studied. Clinical trials are underway to study the combination of various chemotherapeutic agents along with immunotherapy in the management of cholangiocarcinoma, hepatocellular carcinoma, and esophageal cancer. Alternative therapies are needed based on the tumor immune microenvironment, which can lead to a personalized approach to treatment. In this review, we discuss the current status of various modalities of immunotherapy in common GI malignancies, along with their mechanisms of immune activation and cancer suppression. We will also discuss the use of immunotherapy in less common solid GI malignancies and touch on recent advancements and clinical trials. Full article
(This article belongs to the Special Issue Gene and Cell Therapy: New Findings from Medical Research and Treatment)
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