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溶瘤病毒和嵌合抗原受体T细胞基因治疗进展及治疗相关分组

Advances in Gene Therapy with Oncolytic Viruses and CAR-T Cells and Therapy-Related Groups.

作者信息

Matsuzaka Yasunari, Yashiro Ryu

机构信息

Division 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.

Administrative Section of Radiation Protection, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo 187-8551, Japan.

出版信息

Curr Issues Mol Biol. 2025 Apr 10;47(4):268. doi: 10.3390/cimb47040268.

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).

摘要

癌症基因治疗作为一种克服难治性癌症的新治疗方法正引起广泛关注。嵌合抗原受体T细胞(CAR-T)疗法已经取得了显著成果,尤其是在血液肿瘤方面。由于CAR-T细胞能在体内增殖,它们具有只需单次给药的优势。此外,针对复发或难治性疾病,针对CD19阳性急性B淋巴细胞白血病(B-急性淋巴细胞白血病,B-ALL)和弥漫性大B细胞淋巴瘤(DLBCL)的年轻患者,已确立了靶向CD19抗原的CAR-T细胞疗法。除了CAR-T细胞疗法,溶瘤病毒是癌症治疗的一种有前景的方法,一些已在临床使用,其他的也在研究其潜在益处。这些病毒感染并杀死癌细胞,引发免疫反应,帮助身体识别和对抗癌症。溶瘤病毒疗法是一种免疫疗法,它使用经过改造的病毒靶向并摧毁肿瘤细胞,同时潜在地刺激抗肿瘤免疫反应。这些病毒在临床试验中已显示出有前景的活性,一些已被批准用于特定癌症,如黑色素瘤。目前正在进行研究以提高其疗效,将其应用扩展到其他癌症类型,并克服与病毒递送相关的后勤挑战。基因治疗有可能治疗由隐性基因疾病引起的疾病,如囊性纤维化、血友病、肌肉萎缩症和镰状细胞贫血,以及获得性遗传疾病,如癌症和像获得性免疫缺陷综合征(艾滋病)这样的病毒感染。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/37de/12025996/57a91f8bf08f/cimb-47-00268-g001.jpg

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