《生命科学》 2026, 38(4): 651-666

肿瘤的细胞与基因治疗研究进展

苗 瑞¹ , 邢素萍² , 郑颂国^3,4,5 , 孙莲慧^3,4,5,*

¹南京医科大学附属上海一院临床医学院，上海 200080 ²上海交通大学医学院附属第一人民医院疑难疾病精准研究中心，上海 200080 ³上海交通大学医学院细胞和基因治疗研究院，上海 201600 ⁴上海交通大学创新免疫治疗全国重点实验室，上海 201600 ⁵上海交通大学医学院附属松江医院，上海 201600

摘　要：

细胞与基因治疗（cell and gene therapy，CGT）作为一种革命性的治疗方法，通过将改造后的细胞或基因导入患者体内治疗疾病。它基于疾病的分子机制，提供微视角和精准干预，实现个体化治疗。近年临床研究表明，CGT在肿瘤治疗领域表现出巨大潜力。然而，该疗法仍面临耗时长、临床试验规模有限、成本高昂及监管复杂等挑战。本文从CGT的治疗机制出发，概述其技术进展，并结合临床试验探讨其在肿瘤治疗中的应用前景，同时分析CGT发展现状与挑战，旨在为推进该领域研究及扩展其肿瘤适应证提供参考。

通讯作者：孙莲慧 , Email:sunlianhui2009@126.com

Recent advances in cellular and gene therapies for cancer

MIAO Rui¹ , XING Su-Ping² , ZHEGN Song-Guo^3,4,5 , SUN Lian-Hui^3,4,5,*

¹Shanghai General Hospital of Nanjing Medical University, Shanghai 200080, China ²Precision Research Center for Refractory Diseases, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200080, China ³School of Cell and Gene Therapy, Shanghai Jiao Tong University School of Medicine, Shanghai 201600, China ⁴State Key Lab of Innovative Immunotherapy, Shanghai Jiao Tong University, Shanghai 201600, China ⁵Shanghai Songjiang Hospital Affiliated to the Shanghai Jiao Tong University School of Medicine, Shanghai 201600, China

Abstract:

Cell and gene therapy (CGT) represents a frontier technology in biomedicine that has brought revolutionary breakthroughs to the treatment of refractory diseases such as cancer. CGT encompasses two major technological directions: cell therapy and gene therapy. Cell therapy involves the ex vivo modification, expansion, or activation of human cells before reinfusion to exert therapeutic effects. Among these, stem cell therapy can repair tissues and modulate the tumor microenvironment through multilineage differentiation and exosome secretion, while immune cell therapy genetically engineers immune cells to enable precise recognition and elimination of tumor cells. Gene therapy, on the other hand, utilizes vectors (viral or non-viral) to deliver normal genes, editing tools, or therapeutic nucleic acids into target cells, correcting pathogenic genes, silencing aberrant genes, or expressing therapeutic proteins to fundamentally disrupt tumorigenesis and progression, with CRISPR and other gene-editing technologies significantly enhancing treatment precision and efficacy. CGT has demonstrated remarkable clinical value across various tumor types. Hematological malignancies represent the most mature application area for CGT, where CAR-T therapy has been commercialized in lymphoma, leukemia, and multiple myeloma, offering long-term remission and even potential cure for relapsed/refractory patients, while ongoing clinical trials continue to optimize efficacy and safety profiles. Simultaneously, CGT applications are rapidly expanding into solid tumor treatment. Melanoma serves as the benchmark field for immune cell therapy in solid tumors, with breakthrough progress achieved through TIL and CART therapies. In non-small cell lung cancer, CGT combined with targeted and immunotherapeutic approaches continues to break new ground, providing novel options for advanced-stage patients. For ovarian cancer, numerous studies are exploring combination strategies between CGT and emerging technologies such as nanomaterials, gradually overcoming the bottlenecks . The CGT industry has currently entered a rapid development phase. Multiple CGT products have received global regulatory approval, covering hematological malignancies and rare diseases. Technological advances are accelerating continuously, with explosive growth in clinical trial numbers and indications expanding comprehensively from hematological tumors to solid tumors and chronic diseases. Meanwhile, the industrial chain is gradually maturing, with continuous optimization of manufacturing processes and quality control systems, propelling CGT from a ″niche therapy″ toward a ″standard treatment modality″. Despite promising prospects, CGT clinical application still faces multiple challenges: the suppressive tumor microenvironment of solid tumors and target heterogeneity limit therapeutic efficacy; safety concerns such as off-target effects and cytokine release syndrome require stringent management; complex manufacturing processes and high costs result in limited accessibility; and regulatory frameworks, long-term efficacy evaluation, and ethical standards require continuous refinement. These issues represent core bottlenecks constraining large-scale CGT application. CGT has fundamentally transformed the paradigm of cancer treatment, providing entirely novel technological pathways for conquering malignant tumors. Looking ahead, with continuous technological innovation, process optimization, and gradual regulatory system improvement, CGT will achieve greater breakthroughs in solid tumor treatment, drive the adoption of personalized precision medicine, and achieve deep integration with surgery, radiotherapy, chemotherapy, targeted therapy, and immunotherapy, ultimately realizing ″curative treatment″ and bringing greater survival hope to patients worldwide.

Communication Author：SUN Lian-Hui , Email:sunlianhui2009@126.com