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Therapeutic strategies for aberrant splicing in cancer and genetic disorders
Clinical Genetics ( IF 3.5 ) Pub Date : 2024-01-02 , DOI: 10.1111/cge.14478
Wenhua Shi 1, 2, 3 , Jingqun Tang 2, 4 , Juanjuan Xiang 1, 2, 3
Affiliation  

Accurate pre-mRNA splicing is essential for proper protein translation; however, aberrant splicing is commonly observed in the context of cancer and genetic disorders. Notably, in genetic diseases, these splicing abnormalities often play a pivotal role. Substantial challenges persist in accurately identifying and classifying disease-induced aberrant splicing, as well as in development of targeted therapeutic strategies. In this review, we examine prevalent forms of aberrant splicing and explore potential therapeutic approaches aimed at addressing these splicing-related diseases. This summary contributes to a deeper understanding of the complexities about aberrant splicing and provide a foundation for the development of effective therapeutic interventions in the field of genetic disorders and cancer.

中文翻译:

癌症和遗传性疾病中异常剪接的治疗策略

准确的前 mRNA 剪接对于正确的蛋白质翻译至关重要;然而,异常剪接常见于癌症和遗传性疾病中。值得注意的是,在遗传疾病中,这些剪接异常通常起着关键作用。在准确识别和分类疾病引起的异常剪接以及制定靶向治疗策略方面仍然存在巨大挑战。在这篇综述中,我们检查了异常剪接的普遍形式,并探索了旨在解决这些剪接相关疾病的潜在治疗方法。该总结有助于更深入地了解异常剪接的复杂性,并为遗传性疾病和癌症领域开发有效的治疗干预措施奠定基础。
更新日期:2024-01-02
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