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 CAR-T 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 of solid tumor treatment. 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 risks 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 implementation. 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″ for cancer and bringing greater survival hope to patients worldwide.
MIAO et al. (Mon,) studied this question.