Targeting RNA splicing modulation: new perspectives for anticancer strategy?

Abstract The excision of introns from pre-mRNA is a crucial process in the expression of the majority of genes. Alternative splicing allows a single gene to generate diverse mRNA and protein products. Aberrant RNA splicing is recognized as a molecular characteristic present in almost all types of tu...

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Main Authors: Xuemei Lv, Xiaoyu Sun, Yang Gao, Xinyue Song, Xiaoyun Hu, Lang Gong, Li Han, Miao He, Minjie Wei
Format: Article
Language:English
Published: BMC 2025-01-01
Series:Journal of Experimental & Clinical Cancer Research
Subjects:
Online Access:https://doi.org/10.1186/s13046-025-03279-w
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author Xuemei Lv
Xiaoyu Sun
Yang Gao
Xinyue Song
Xiaoyun Hu
Lang Gong
Li Han
Miao He
Minjie Wei
author_facet Xuemei Lv
Xiaoyu Sun
Yang Gao
Xinyue Song
Xiaoyun Hu
Lang Gong
Li Han
Miao He
Minjie Wei
author_sort Xuemei Lv
collection DOAJ
description Abstract The excision of introns from pre-mRNA is a crucial process in the expression of the majority of genes. Alternative splicing allows a single gene to generate diverse mRNA and protein products. Aberrant RNA splicing is recognized as a molecular characteristic present in almost all types of tumors. Therefore, identifying cancer-specific subtypes from aberrant processing offers new opportunities for therapeutic development. Numerous splicing modulators, each utilizing different mechanisms, have been developed as promising anticancer therapies, some of which are in clinical trials. In this review, we summarize the splice-altered signatures of cancer cell transcriptomes and the contributions of splicing aberrations to tumorigenesis and progression. Especially, we discuss current and emerging RNA splicing-targeted strategies for cancer therapy, including pharmacological approaches and splice-switching antisense oligonucleotides (ASOs). Finally, we address the challenges and opportunities in translating these findings into clinical practice.
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institution Kabale University
issn 1756-9966
language English
publishDate 2025-01-01
publisher BMC
record_format Article
series Journal of Experimental & Clinical Cancer Research
spelling doaj-art-e7b8934eea404f749e0a56f0f0dc427d2025-02-02T12:47:45ZengBMCJournal of Experimental & Clinical Cancer Research1756-99662025-01-0144112210.1186/s13046-025-03279-wTargeting RNA splicing modulation: new perspectives for anticancer strategy?Xuemei Lv0Xiaoyu Sun1Yang Gao2Xinyue Song3Xiaoyun Hu4Lang Gong5Li Han6Miao He7Minjie Wei8Department of Pharmacology, School of Pharmacy, China Medical UniversityDepartment of Pharmacology, School of Pharmacy, China Medical UniversityDepartment of Pharmacology, School of Pharmacy, China Medical UniversityDepartment of Pharmacology, School of Pharmacy, China Medical UniversityScientific Experimental Center, School of Pharmacy, China Medical UniversityDepartment of Pharmacology, School of Pharmacy, China Medical UniversityDepartment of Pharmacology, School of Pharmacy, China Medical UniversityDepartment of Pharmacology, School of Pharmacy, China Medical UniversityDepartment of Pharmacology, School of Pharmacy, China Medical UniversityAbstract The excision of introns from pre-mRNA is a crucial process in the expression of the majority of genes. Alternative splicing allows a single gene to generate diverse mRNA and protein products. Aberrant RNA splicing is recognized as a molecular characteristic present in almost all types of tumors. Therefore, identifying cancer-specific subtypes from aberrant processing offers new opportunities for therapeutic development. Numerous splicing modulators, each utilizing different mechanisms, have been developed as promising anticancer therapies, some of which are in clinical trials. In this review, we summarize the splice-altered signatures of cancer cell transcriptomes and the contributions of splicing aberrations to tumorigenesis and progression. Especially, we discuss current and emerging RNA splicing-targeted strategies for cancer therapy, including pharmacological approaches and splice-switching antisense oligonucleotides (ASOs). Finally, we address the challenges and opportunities in translating these findings into clinical practice.https://doi.org/10.1186/s13046-025-03279-wCancerAlternative splicingSplicing modulatorsSplice-switching ASOTargeted therapy
spellingShingle Xuemei Lv
Xiaoyu Sun
Yang Gao
Xinyue Song
Xiaoyun Hu
Lang Gong
Li Han
Miao He
Minjie Wei
Targeting RNA splicing modulation: new perspectives for anticancer strategy?
Journal of Experimental & Clinical Cancer Research
Cancer
Alternative splicing
Splicing modulators
Splice-switching ASO
Targeted therapy
title Targeting RNA splicing modulation: new perspectives for anticancer strategy?
title_full Targeting RNA splicing modulation: new perspectives for anticancer strategy?
title_fullStr Targeting RNA splicing modulation: new perspectives for anticancer strategy?
title_full_unstemmed Targeting RNA splicing modulation: new perspectives for anticancer strategy?
title_short Targeting RNA splicing modulation: new perspectives for anticancer strategy?
title_sort targeting rna splicing modulation new perspectives for anticancer strategy
topic Cancer
Alternative splicing
Splicing modulators
Splice-switching ASO
Targeted therapy
url https://doi.org/10.1186/s13046-025-03279-w
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