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Updated: Sep 14, 2025

A Nonviral Approach to Generate Transient Chimeric Antigen Receptor T Cells Using mRNA for Cancer Immunotherapy
Published on: February 21, 2025
Advances in Gene Therapy with Oncolytic Viruses and CAR-T Cells and Therapy-Related Groups
Yasunari Matsuzaka1,2, Ryu Yashiro2,3
1Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, Minato-ku, Tokyo 108-8639, Japan.
Abstract:
Cancer gene therapy is attracting considerable attention as a new treatment method for overcoming intractable cancers. CAR-T cell therapy has already achieved remarkable results, particularly for hematological tumors. Because CAR-T cells can increase within the body, they have the advantage of requiring only a single administration. In addition, CAR-T cell therapy targeting the CD19 antigen has been established for relapsed or refractory disease in young people with CD19-positive acute B-cell leukemia (B-acute lymphoblastic leukemia, B-ALL) and diffuse large B-cell lymphoma (DLBCL). In addition to CAR-T cell therapy, oncolytic viruses represent a promising approach for cancer treatment, with some already in clinical use and others being researched for their potential benefits. These viruses infect and kill cancer cells, triggering an immune response that helps the body recognize and fight cancer. Oncolytic virus therapy is a form of immunotherapy that uses modified viruses to target and destroy tumor cells while potentially stimulating antitumor immune responses. These viruses have shown promising activity in clinical trials, with some approved for specific cancers like melanoma. Research is ongoing to improve their efficacy, expand their use to other cancer types, and overcome the logistical challenges associated with their delivery. Gene therapy can potentially treat diseases caused by recessive gene disorders like cystic fibrosis, hemophilia, muscular dystrophy, and sickle cell anemia, as well as acquired genetic diseases, such as cancer and viral infections like acquired immunodeficiency syndrome (AIDS).
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