Advances in Cell and Gene Therapy in Tumors: From Bench to Bedside

A special issue of Cancers (ISSN 2072-6694). This special issue belongs to the section "Cancer Therapy".

Deadline for manuscript submissions: 31 December 2025 | Viewed by 537

Special Issue Editor


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Guest Editor Assistant
Clinical Cancer Center, National Cancer Center/National Clinical Research Center for Cancer, Cancer Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing 100005, China
Interests: cell therapy; gene therapy; tumor microenvironment; immunotherapy; oncogenomics; targeted therapy; personalized medicine; cancer drug development; translational research; clinical trials

Special Issue Information

Dear Colleagues,

Cell-based therapy, which involves the administration of therapeutic cells as living agents to treat diseases, has currently experienced explosive growth in both pre-clinical development and expansion within the pharmaceutical marketplace. Adoptive cell therapy (ACT) takes advantage of immune cells to eliminate tumors. ACT can be broadly classified as non-genetically and genetically engineered cell products or more specifically based on the cell type, such as T cells, natural killer (NK) cells, dendritic cells (DCs), cytokine-induced killer (CIK) cells, tumor-infiltrating lymphocytes (TILs), and mesenchymal stromal cells (MSCs). Both non-genetically and genetically engineered ACTs are either available or being actively investigated in clinical settings involving T cells (CAR-T, TCR-T, and γδ T cells, as well as gene-edited T cells, respectively), TILs, DCs, and macrophages. Meanwhile, gene therapy involves the use of nucleic acids (DNA or RNA) for the treatment of various human diseases. Based on the pathogenesis mechanism, the ideal gene therapy can be achieved either by delivering a therapeutic gene as a substitute for the missing or defective endogenous counterpart or by reducing the levels of a harmful defective gene product, using naked oligonucleotides, as well as viral and non-viral vectors. Genetically engineered cells also belong to the category of gene therapy, using extraneous genetic products to treat diseases such as tumors. Many pre-clinical explorations and clinical trials have exhibited the promising potential of cell and gene therapies in the treatment of human tumors. From 1 January 2010 to 31 December 2019, 491 clinical trials comprising a total of 178 cell and gene therapies were carried out worldwide (INFORMA database). However, cell and gene therapeutics are an emerging modality with the potential to treat many currently intractable diseases, especially tumors, through uniquely powerful modes of action. Despite notable recent clinical and commercial successes, cell and gene therapies continue to face numerous challenges that limit their widespread translation and commercialization, including the identification of an appropriate cell source and gene vehicle, the generation of a sufficiently viable, potent, and safe product that meets patient- and disease-specific needs, and the development of scalable manufacturing processes. These hurdles are being addressed through the use of cutting-edge basic research, like genome and epigenome editing, advanced biomaterials, and so on. The goal of this Special Issue is to explore the potential of cell and gene therapy for the treatment of human tumors, aiming to bring together original research, reviews, perspectives, comments, and letters to editors that cover the latest advances and challenges in the development and application of cell and gene therapy in tumors. Potential subtopics include but are not limited to the following: 

  • the pre-clinical study of cell and gene therapy in tumors;
  • the clinical application and management of cell and gene therapy in tumors;
  • next-generation cell and gene therapy development;
  • clinical trials of cell and gene therapy in human tumors;
  • polices and guidelines of cell and gene therapy.

Dr. Shuhang Wang
Guest Editor

Manuscript Submission Information

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Keywords

  • cell therapy
  • gene therapy
  • genetically engineered cells
  • cancer treatment
  • clinical trials
  • pre-clinical study

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Published Papers (1 paper)

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Research

28 pages, 15046 KB  
Article
Application of Single-Cell Sequencing and Machine Learning in Prognosis and Immune Profiling of Lung Adenocarcinoma: Exploring Disease Mechanisms and Treatment Strategies Based on Circadian Rhythm Gene Signatures
by Qiuqiao Mu, Han Zhang, Kai Wang, Lin Tan, Xin Li and Daqiang Sun
Cancers 2025, 17(17), 2911; https://doi.org/10.3390/cancers17172911 - 5 Sep 2025
Abstract
Background: The circadian rhythm regulates important functions in the body, such as metabolism, the cell cycle, DNA repair, and immune balance. Disruption of this rhythm can contribute to the development of cancer. Circadian rhythm genes (CRGs) are attracting attention for their connection [...] Read more.
Background: The circadian rhythm regulates important functions in the body, such as metabolism, the cell cycle, DNA repair, and immune balance. Disruption of this rhythm can contribute to the development of cancer. Circadian rhythm genes (CRGs) are attracting attention for their connection to various cancers. However, their roles in LUAD are not yet well understood. Additionally, our knowledge of how they function at both the bulk tissue and single-cell levels is limited. This gap hinders a complete understanding of how CRGs impact the development and outcomes of LUAD. Methods: We selected 554 CRGs from public databases. We then obtained transcriptome data from TCGA and GEO. A total of 101 machine learning algorithm combinations were tested using 10 algorithms and 10-fold cross-validation. The best-performing model was based on Stepwise Cox regression and SuperPC. This model was validated with additional datasets. We also examined the relationships between CRGs, immune features, tumor mutation burden (TMB), and the response to immunotherapy. Drug sensitivity was also assessed. Single-cell data identified the cell types with active CRGs. Next, we performed qRT-PCR and other basic experiments to validate the expression of ARNTL2 in LUAD tissues and cell lines. The results indicated that ARNTL2 may play a key role in lung adenocarcinoma. Results: The CRG-based model clearly distinguished LUAD patients based on their risk. High-risk patients exhibited low immune activity, high TMB, and poor predicted responses to immunotherapy. Single-cell data revealed strong CRG signals in epithelial and fibroblast cells. These cell groups also displayed different communication patterns. Laboratory experiments showed that ARNTL2 was highly expressed in LUAD. It promoted cell growth, movement, and invasion. This suggests that ARNTL2 may play a role in promoting cancer. Conclusions: This study developed a machine learning model based on CRGs. It can predict survival and immune status in LUAD patients. The research also identified ARNTL2 as a key gene that may contribute to cancer progression. These findings highlight the significance of the circadian rhythm in LUAD and provide new perspectives for diagnosis and treatment. Full article
(This article belongs to the Special Issue Advances in Cell and Gene Therapy in Tumors: From Bench to Bedside)
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