A Dual Approach with Organoid and CRISPR Screening Reveals ERCC6 as a Determinant of Cisplatin Resistance in Osteosarcoma

Abstract Osteosarcoma (OS), the most prevalent primary bone malignancy in adolescents, is typically treated with cisplatin‐based chemotherapy. However, the development of cisplatin resistance often leads to relapse or metastasis, significantly impairing therapeutic efficacy. To tackle this issue, pa...

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Main Authors: Ruiling Xu, Sai Zhu, Wenchao Zhang, Haodong Xu, Chao Tu, Honghui Wang, Lu Wang, Na He, Tang Liu, Xiaoning Guo, Xiaolei Ren, Zhihong Li
Format: Article
Language:English
Published: Wiley 2025-07-01
Series:Advanced Science
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Online Access:https://doi.org/10.1002/advs.202500632
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author Ruiling Xu
Sai Zhu
Wenchao Zhang
Haodong Xu
Chao Tu
Honghui Wang
Lu Wang
Na He
Tang Liu
Xiaoning Guo
Xiaolei Ren
Zhihong Li
author_facet Ruiling Xu
Sai Zhu
Wenchao Zhang
Haodong Xu
Chao Tu
Honghui Wang
Lu Wang
Na He
Tang Liu
Xiaoning Guo
Xiaolei Ren
Zhihong Li
author_sort Ruiling Xu
collection DOAJ
description Abstract Osteosarcoma (OS), the most prevalent primary bone malignancy in adolescents, is typically treated with cisplatin‐based chemotherapy. However, the development of cisplatin resistance often leads to relapse or metastasis, significantly impairing therapeutic efficacy. To tackle this issue, patient‐derived osteosarcoma organoids (OSOs) is established that accurately reflect the cellular composition and heterogeneity of the original tumors, as validated by single‐cell RNA sequencing, bulk RNA sequencing, and histopathology analysis. Cisplatin resistance is successfully induced in these OSOs, creating a clinically relevant model for investigating chemoresistance. Utilizing RNA sequencing in cisplatin‐resistance OSOs and CRISPR screening in OS cell line, ERCC6 is identified as a pivotal regulator of cisplatin resistance. Knockdown of ERCC6 markedly enhanced cisplatin sensitivity in vitro and in vivo. Mechanistically, ERCC6 interacts with HNRNPM, influencing the PI3K/AKT signaling pathway and alternative splicing of pre‐mRNA for BAX. Notably, the knockdown of ERCC6 and HNRNPM increased expression of full‐length BAX and reduced skipping of exon 2, thus promoting apoptosis. This exon skipping in BAX results in a frameshift and introduces a premature stop codon (TGA) within the BH3 domain. These findings underscore the utility of OSOs in elucidating resistance mechanisms and highlight ERCC6 and HNRNPM as potential therapeutic targets.
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spelling doaj-art-898a8a25b28b4a6a9de6142b52b358292025-08-20T03:32:37ZengWileyAdvanced Science2198-38442025-07-011228n/an/a10.1002/advs.202500632A Dual Approach with Organoid and CRISPR Screening Reveals ERCC6 as a Determinant of Cisplatin Resistance in OsteosarcomaRuiling Xu0Sai Zhu1Wenchao Zhang2Haodong Xu3Chao Tu4Honghui Wang5Lu Wang6Na He7Tang Liu8Xiaoning Guo9Xiaolei Ren10Zhihong Li11Department of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaDepartment of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaDepartment of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaDepartment of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaDepartment of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaCollege of Biology Hunan University Changsha 410082 P. R. ChinaDepartment of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaDepartment of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaDepartment of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaDepartment of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaDepartment of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaDepartment of Orthopedics The Second Xiangya Hospital Central South University Changsha Hunan 410011 P. R. ChinaAbstract Osteosarcoma (OS), the most prevalent primary bone malignancy in adolescents, is typically treated with cisplatin‐based chemotherapy. However, the development of cisplatin resistance often leads to relapse or metastasis, significantly impairing therapeutic efficacy. To tackle this issue, patient‐derived osteosarcoma organoids (OSOs) is established that accurately reflect the cellular composition and heterogeneity of the original tumors, as validated by single‐cell RNA sequencing, bulk RNA sequencing, and histopathology analysis. Cisplatin resistance is successfully induced in these OSOs, creating a clinically relevant model for investigating chemoresistance. Utilizing RNA sequencing in cisplatin‐resistance OSOs and CRISPR screening in OS cell line, ERCC6 is identified as a pivotal regulator of cisplatin resistance. Knockdown of ERCC6 markedly enhanced cisplatin sensitivity in vitro and in vivo. Mechanistically, ERCC6 interacts with HNRNPM, influencing the PI3K/AKT signaling pathway and alternative splicing of pre‐mRNA for BAX. Notably, the knockdown of ERCC6 and HNRNPM increased expression of full‐length BAX and reduced skipping of exon 2, thus promoting apoptosis. This exon skipping in BAX results in a frameshift and introduces a premature stop codon (TGA) within the BH3 domain. These findings underscore the utility of OSOs in elucidating resistance mechanisms and highlight ERCC6 and HNRNPM as potential therapeutic targets.https://doi.org/10.1002/advs.202500632alternative splicingchemoresistanceERCC6organoidosteosarcoma
spellingShingle Ruiling Xu
Sai Zhu
Wenchao Zhang
Haodong Xu
Chao Tu
Honghui Wang
Lu Wang
Na He
Tang Liu
Xiaoning Guo
Xiaolei Ren
Zhihong Li
A Dual Approach with Organoid and CRISPR Screening Reveals ERCC6 as a Determinant of Cisplatin Resistance in Osteosarcoma
Advanced Science
alternative splicing
chemoresistance
ERCC6
organoid
osteosarcoma
title A Dual Approach with Organoid and CRISPR Screening Reveals ERCC6 as a Determinant of Cisplatin Resistance in Osteosarcoma
title_full A Dual Approach with Organoid and CRISPR Screening Reveals ERCC6 as a Determinant of Cisplatin Resistance in Osteosarcoma
title_fullStr A Dual Approach with Organoid and CRISPR Screening Reveals ERCC6 as a Determinant of Cisplatin Resistance in Osteosarcoma
title_full_unstemmed A Dual Approach with Organoid and CRISPR Screening Reveals ERCC6 as a Determinant of Cisplatin Resistance in Osteosarcoma
title_short A Dual Approach with Organoid and CRISPR Screening Reveals ERCC6 as a Determinant of Cisplatin Resistance in Osteosarcoma
title_sort dual approach with organoid and crispr screening reveals ercc6 as a determinant of cisplatin resistance in osteosarcoma
topic alternative splicing
chemoresistance
ERCC6
organoid
osteosarcoma
url https://doi.org/10.1002/advs.202500632
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