RNA剪接失调被认为是癌症的分子标志和重要治疗靶点。虽然靶向剪接已被广泛用于癌症治疗，但普遍缺乏针对失调剪接因子的特异性调节剂。 RNA 结合基序蛋白 10 (RBM10) 在多种癌症中经常发生突变，特别是肺腺癌。越来越多的证据表明，由于功能丧失突变或异常表达而导致的 RBM10 缺陷会导致癌症的发生、进展和治疗反应。在这里，我们总结了 RBM10 缺乏对癌症的功能影响和新的治疗意义。以 RBM10 作为代表性例子，我们重点介绍了主要策略并讨论了针对癌症治疗的失调剪接因子的考虑因素。这些策略单独或与目前批准的疗法相结合，可能在癌症治疗中具有广阔的前景。© 作者 2023。由牛津大学出版社代表中国科学院上海生命科学研究院生物化学与细胞生物学研究所出版科学。

RNA splicing dysregulation is considered as a molecular hallmark and important therapeutic target of cancer. While targeting splicing has been intensively pursued for cancer therapy, specific modulators for dysregulated splicing factors are generally lacking. RNA binding motif protein 10 (RBM10) is frequently mutated in multiple cancers, in particular lung adenocarcinoma. Increasing evidence demonstrates that RBM10 deficiency due to loss-of-function mutation or aberrant expression contributes to cancer development, progression and therapy response. Here we summarize the functional consequences and new therapeutic implications of RBM10 deficiency in cancer. Using RBM10 as a representative example, we highlight the main strategies and discuss the considerations of targeting dysregulated splicing factors for cancer therapy. Those strategies alone or in combination with currently approved therapies may hold great promise in cancer treatment.© The Author(s) 2023. Published by Oxford University Press on behalf of Institute of Biochemistry and Cell Biology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences.