Page 29 - 《中国药科大学学报》2026年第2期

学报
Journal of China Pharmaceutical University 2026, 57(2): 155 − 162 155

RNA 可变剪接机制及其在肿瘤治疗中的研究进展

黄昕欣 ，唐铭珠 ，王多伟 1,2*
1,2
1,2
2
（ 中国药科大学基础医学与临床药学学院, 南京 211198； 中国药科大学转化医学研究院, 南京 210009）
1
摘 要 RNA 可变剪接（alternative splicing，AS）是基因表达调控的关键机制，通过产生多种 mRNA 剪接异构体，增加蛋白
质的多样性，对细胞生理过程如增殖、代谢、凋亡等至关重要。在肿瘤治疗领域，可变剪接的研究进展显著，异常的可变剪接
参与调控肿瘤发生、发展的多种生物学过程。研究表明，特定剪接因子的突变或表达变化在多种癌症中起关键作用，如
SF3B1、SRSF2、U2AF1 等。在此基础上，本文总结了针对可变剪接的肿瘤治疗新策略，包括抑制剪接体组分、反义寡核苷酸
（ASO）疗法以及克服肿瘤耐药性和免疫逃逸的方法，这些策略通过调控剪接因子和修饰酶的活性，展现出克服传统化疗耐
药问题和增强免疫治疗效果的潜力。本综述从可变剪接的调控机制、在肿瘤中的作用及其在肿瘤治疗中的新策略和应用前
景出发，全面探讨可变剪接在肿瘤研究和治疗中的重要性。
关键词 可变剪接；剪接体；肿瘤治疗；靶向药物；反义寡核苷酸疗法；耐药治疗
中图分类号 R730.5 文献标志码 A 文章编号 1000−5048(2026)02−0155−08
doi：10.11665/j.issn.1000−5048.2005022401

引用本文 黄昕欣，唐铭珠，王多伟. RNA 可变剪接机制及其在肿瘤治疗中的研究进展 [J]. 中国药科大学学报，2026，57（2）：155-162.

Cite this article as: HUANG Xinxin, TANG Mingzhu, WANG Duowei. Research progress of RNA alternative splicing mechanisms and their
roles in cancer therapy[J]. J China Pharm Univ, 2026, 57(2): 155-162.

Research progress of RNA alternative splicing mechanisms and their roles in
cancer therapy

1,2
1,2
HUANG Xinxin , TANG Mingzhu , WANG Duowei 1,2*
1
School of Basic Medical Sciences and Clinical Pharmacy, China Pharmaceutical University, Nanjing 211198;
2
Institute of Translational Medicine, China Pharmaceutical University, Nanjing 210009, China
Abstract Alternative splicing (AS), a critical mechanism in gene expression regulation, is essential for cellular
physiological processes such as proliferation, metabolism, and apoptosis via generating diverse mRNA isoforms
and expanding protein diversity. In the field of oncology, AS has been significantly implicated in the regulation of
various biological processes involved in tumorigenesis and cancer progression. Specific mutations or expression
changes in splicing factors, such as SF3B1, SRSF2, and U2AF1, play key roles in multiple types of cancer. This
review summarizes novel therapeutic strategies for cancer targeting alternative splicing, including inhibiting
spliceosome components, antisense oligonucleotide (ASO) therapy, and approaches to overcome drug resistance
and immune evasion in cancer. These strategies, which modulate the activity of splicing factors and RNA-
modifying enzymes, show promise in addressing chemoresistance and enhancing the efficacy of immunotherapy.
This review provides a comprehensive exploration of alternative splicing mechanisms, their role in cancer, and
their potential as a therapeutic target, highlighting the importance of alternative splicing in cancer research and
therapy.
Key words alternative splicing; spliceosome; cancer therapy; targeted drugs; antisense oligonucleotide
therapy; drug resistance therapy

精确的基因表达调控对于生物体的发育过程 录时，外显子和内含子一起被转录为前体 mRNA
和细胞正常生理功能至关重要。真核生物基因转 （pre-mRNA）。该过程可概括为两个主要的步骤，

收稿日期 2005-02-24 * 通信作者 E-mail：wangduowei@cpu.edu.cn