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Immune Cell Therapy and Genome Engineering

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".

Deadline for manuscript submissions: closed (20 April 2026) | Viewed by 1254

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Department of Interdisciplinary Medicine, School of Medicine, University of Bari “Aldo Moro”, 70124 Bari, Italy
Interests: mitochondria; cancer; chronic diseases; infectious disease
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Immune cell therapies, such as CAR-T cells, TCR-T cells, and NK cell therapies, are transforming the landscape of cancer treatment and chronic immune disorders. These therapies harness and reprogram the patient’s immune cells to recognize, target, and eliminate disease-causing cells. Beyond oncology, immune cell therapies are showing promise in infectious diseases and autoimmune conditions, offering a highly personalized therapeutic approach.

Regenerative therapy seeks to repair or replace damaged cells, tissues, and organs using stem cells, biomaterials, and bioengineered constructs. Innovations in mesenchymal stem cell (MSC) therapy, platelet-rich plasma (PRP), exosome therapies, and tissue engineering are redefining possibilities for healing and recovery in degenerative, inflammatory, and trauma-related conditions. These strategies hold significant promise for neurodegeneration, cardiovascular repair, musculoskeletal restoration, and beyond.

Advancements in gene editing technologies, such as CRISPR-Cas9, base editors, and prime editors, allow precise alterations in the genome to correct genetic defects or modulate disease pathways. These tools are rapidly being integrated into therapeutic pipelines for monogenic diseases, oncology, infectious diseases, and even for enhancing immune cell therapies. The combination of gene editing with cell-based platforms offers a powerful modality for curative interventions.

Understanding gene regulation at the epigenetic level—through DNA methylation, histone modifications, non-coding RNAs, and chromatin architecture—adds a crucial layer to precision medicine. Epigenetic modulation can influence disease susceptibility, therapeutic response, and aging processes. The emergence of epigenome-editing tools allows targeted modulation of gene expression without altering the underlying DNA sequence, offering reversible and programmable control of cellular behavior.

This Special Issue aims to highlight the integration of cutting-edge innovations in immune modulation, regeneration, genome engineering, and epigenetic reprogramming—ushering in a new era of systems-level, personalized, and regenerative medicine. These interwoven disciplines not only enhance therapeutic precision but also redefine the boundaries of what is biologically and clinically possible.

Prof. Dr. Kieu C. D. Nguyễn
Guest Editor

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Keywords

  • immune cell therapies
  • regenerative therapy
  • gene editing technologies
  • epigenetic modulation
  • precision medicine
  • personalized therapy

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Published Papers (1 paper)

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Research

12 pages, 1461 KB  
Article
Clinical Impact of LAG3 Single-Nucleotide Polymorphism in DLBCL Treated with CAR-T Cell Therapy
by Katja Seipel, Sophia Maria Spahr, Inna Shaforostova, Ulrike Bacher, Henning Nilius and Thomas Pabst
Int. J. Mol. Sci. 2025, 26(20), 9905; https://doi.org/10.3390/ijms26209905 - 11 Oct 2025
Cited by 4 | Viewed by 873
Abstract
Lymphocyte-activation gene 3 (LAG3) is an immune checkpoint receptor and inhibitory regulator of T-cells. Here, we analyzed the prevalence of LAG3 rs870849 in B-cell lymphoma patients and the treatment outcomes according to the LAG3 genetic background and discovered that LAG3 germline [...] Read more.
Lymphocyte-activation gene 3 (LAG3) is an immune checkpoint receptor and inhibitory regulator of T-cells. Here, we analyzed the prevalence of LAG3 rs870849 in B-cell lymphoma patients and the treatment outcomes according to the LAG3 genetic background and discovered that LAG3 germline variants may affect the risk of developing lymphoma and also affect the treatment outcome of DLBCL patients in the current CD19 CAR-T cell therapies. The LAG3 rs870849 was prevalent at high frequency in DLBCL patients. Significant differences in treatment outcomes to CAR-T cell therapy emerged in LAG3 I455hom versus I455Thet and T455hom carriers. The overall and complete response rates to CAR-T cell therapy were lower in the I455hom genetic subgroup with median PFS in the I455hom of 2 versus 20 months in the T455hom and I455Thet subgroups (p = 0.025). Median OS was 6 months in the LAG3 I455hom versus 41 months in the T455hom and I455Thet subgroups (p = 0.007). LAG3 rs870849 may affect treatment outcome in CAR-T cell therapy, with favorable outcomes in T455 carriers. Specific combinations of CTLA4 and LAG3 germline variants may cooperate to affect the response to CAR-T cell therapy. Full article
(This article belongs to the Special Issue Immune Cell Therapy and Genome Engineering)
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