CRISPR/Cas9-based discovery of ccRCC therapeutic opportunities through molecular mechanism and immune microenvironment analysis

IntroductionClear cell renal cell carcinoma is a common and aggressive form of renal cell carcinoma. Its incidence continues to rise, and metastatic recurrence leads to poor clinical outcomes. Current prognostic biomarkers lack reliability. We integrated multi-omics data to discover key ccRCC genes...

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Main Authors: Bo Han, Weiyang Liu, Wanhui Wang, Zhuolun Li, Bosen You, Dongze Liu, Yunfeng Nan, Tiankai Ding, Zhou Dai, Yantong Zhang, Wei Zhang, Qing Liu, Xuedong Li
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-07-01
Series:Frontiers in Immunology
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Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2025.1619361/full
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author Bo Han
Weiyang Liu
Wanhui Wang
Zhuolun Li
Bosen You
Dongze Liu
Yunfeng Nan
Tiankai Ding
Zhou Dai
Yantong Zhang
Wei Zhang
Qing Liu
Xuedong Li
author_facet Bo Han
Weiyang Liu
Wanhui Wang
Zhuolun Li
Bosen You
Dongze Liu
Yunfeng Nan
Tiankai Ding
Zhou Dai
Yantong Zhang
Wei Zhang
Qing Liu
Xuedong Li
author_sort Bo Han
collection DOAJ
description IntroductionClear cell renal cell carcinoma is a common and aggressive form of renal cell carcinoma. Its incidence continues to rise, and metastatic recurrence leads to poor clinical outcomes. Current prognostic biomarkers lack reliability. We integrated multi-omics data to discover key ccRCC genes and build a prognostic model to improve risk prediction and guide treatment decisions.MethodsOur study integrated genome-wide CRISPR screening data from DepMap and transcriptomic profiles from TCGA to identify key genes associated with ccRCC pathogenesis. Initial screening identified 11 candidate genes through differential expression analysis and CRISPR functional validation. Using LASSO and Cox regression, we selected five key genes (GGT6, HAO2, SLPI, MELK, and EIF4A1) for model construction. The functional role of MELK was tested by knockdown experiments. Additional analyses included tumor mutation burden, immune microenvironment assessment, and drug response prediction.ResultsThe model stratified patients into high-risk and low-risk groups with distinct survival outcomes. High-risk cases showed higher mutation loads, immunosuppressive features, and activated cytokine pathways, whereas low-risk cases displayed metabolic pathway activity. MELK knockdown reduced cancer cell proliferation and migration. High-risk patients exhibited better responses to targeted drugs such as pazopanib and sunitinib.DiscussionOur study demonstrates the pivotal role of MELK in ccRCC progression. This multi-omics-driven model elucidates MELK-mediated mechanisms and their interactions with the tumor microenvironment, providing novel strategies for risk stratification and targeted therapy. Future studies will validate these findings in independent cohorts and investigate the regulatory networks of MELK to identify potential therapeutic targets.
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spelling doaj-art-86fefec0b49f4ba3b06b07ced8a538f52025-08-20T03:28:50ZengFrontiers Media S.A.Frontiers in Immunology1664-32242025-07-011610.3389/fimmu.2025.16193611619361CRISPR/Cas9-based discovery of ccRCC therapeutic opportunities through molecular mechanism and immune microenvironment analysisBo HanWeiyang LiuWanhui WangZhuolun LiBosen YouDongze LiuYunfeng NanTiankai DingZhou DaiYantong ZhangWei ZhangQing LiuXuedong LiIntroductionClear cell renal cell carcinoma is a common and aggressive form of renal cell carcinoma. Its incidence continues to rise, and metastatic recurrence leads to poor clinical outcomes. Current prognostic biomarkers lack reliability. We integrated multi-omics data to discover key ccRCC genes and build a prognostic model to improve risk prediction and guide treatment decisions.MethodsOur study integrated genome-wide CRISPR screening data from DepMap and transcriptomic profiles from TCGA to identify key genes associated with ccRCC pathogenesis. Initial screening identified 11 candidate genes through differential expression analysis and CRISPR functional validation. Using LASSO and Cox regression, we selected five key genes (GGT6, HAO2, SLPI, MELK, and EIF4A1) for model construction. The functional role of MELK was tested by knockdown experiments. Additional analyses included tumor mutation burden, immune microenvironment assessment, and drug response prediction.ResultsThe model stratified patients into high-risk and low-risk groups with distinct survival outcomes. High-risk cases showed higher mutation loads, immunosuppressive features, and activated cytokine pathways, whereas low-risk cases displayed metabolic pathway activity. MELK knockdown reduced cancer cell proliferation and migration. High-risk patients exhibited better responses to targeted drugs such as pazopanib and sunitinib.DiscussionOur study demonstrates the pivotal role of MELK in ccRCC progression. This multi-omics-driven model elucidates MELK-mediated mechanisms and their interactions with the tumor microenvironment, providing novel strategies for risk stratification and targeted therapy. Future studies will validate these findings in independent cohorts and investigate the regulatory networks of MELK to identify potential therapeutic targets.https://www.frontiersin.org/articles/10.3389/fimmu.2025.1619361/fullCRISPR-Cas9 screeningccRCCprognostic modelMELKimmunotherapy
spellingShingle Bo Han
Weiyang Liu
Wanhui Wang
Zhuolun Li
Bosen You
Dongze Liu
Yunfeng Nan
Tiankai Ding
Zhou Dai
Yantong Zhang
Wei Zhang
Qing Liu
Xuedong Li
CRISPR/Cas9-based discovery of ccRCC therapeutic opportunities through molecular mechanism and immune microenvironment analysis
Frontiers in Immunology
CRISPR-Cas9 screening
ccRCC
prognostic model
MELK
immunotherapy
title CRISPR/Cas9-based discovery of ccRCC therapeutic opportunities through molecular mechanism and immune microenvironment analysis
title_full CRISPR/Cas9-based discovery of ccRCC therapeutic opportunities through molecular mechanism and immune microenvironment analysis
title_fullStr CRISPR/Cas9-based discovery of ccRCC therapeutic opportunities through molecular mechanism and immune microenvironment analysis
title_full_unstemmed CRISPR/Cas9-based discovery of ccRCC therapeutic opportunities through molecular mechanism and immune microenvironment analysis
title_short CRISPR/Cas9-based discovery of ccRCC therapeutic opportunities through molecular mechanism and immune microenvironment analysis
title_sort crispr cas9 based discovery of ccrcc therapeutic opportunities through molecular mechanism and immune microenvironment analysis
topic CRISPR-Cas9 screening
ccRCC
prognostic model
MELK
immunotherapy
url https://www.frontiersin.org/articles/10.3389/fimmu.2025.1619361/full
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