RNA错配剪接是几乎所有肿瘤类型的分子特征。癌症相关的剪接变化源自于反复突变和调控剪接催化和调控的转作用因子表达的改变。癌症相关的剪接调节失控可以通过多种机制促进肿瘤发生和发展，如增加细胞增殖、减少细胞凋亡、增强迁移和转移潜力、耐药性以及免疫逃避。最近的研究已经确认了特定的癌症相关同型体在癌细胞转化和生长中发挥关键作用，并证明了矫正或拮抗这种癌症相关的mRNA同型体的治疗效益。临床级别的小分子药物可以调节或抑制RNA剪接，也已被开发为有前途的抗癌治疗药物。在这里，我们回顾了与癌细胞转录组特征相关的剪接变化、失调的剪接对肿瘤初始化和进展的贡献以及现有和新兴的针对剪接的肿瘤治疗方法。最后，我们讨论了需要解决的突出问题和挑战，以将这些发现转化为临床应用。© 2023. Springer Nature Limited.

Dysregulated RNA splicing is a molecular feature that characterizes almost all tumour types. Cancer-associated splicing alterations arise from both recurrent mutations and altered expression of trans-acting factors governing splicing catalysis and regulation. Cancer-associated splicing dysregulation can promote tumorigenesis via diverse mechanisms, contributing to increased cell proliferation, decreased apoptosis, enhanced migration and metastatic potential, resistance to chemotherapy and evasion of immune surveillance. Recent studies have identified specific cancer-associated isoforms that play critical roles in cancer cell transformation and growth and demonstrated the therapeutic benefits of correcting or otherwise antagonizing such cancer-associated mRNA isoforms. Clinical-grade small molecules that modulate or inhibit RNA splicing have similarly been developed as promising anticancer therapeutics. Here, we review splicing alterations characteristic of cancer cell transcriptomes, dysregulated splicing's contributions to tumour initiation and progression, and existing and emerging approaches for targeting splicing for cancer therapy. Finally, we discuss the outstanding questions and challenges that must be addressed to translate these findings into the clinic.© 2023. Springer Nature Limited.