New microRNA-based therapies reveal common targets in paediatric medulloblastoma and adult glioblastoma

Denis  Mustafov; Laura Thomas; Shoib Siddiqui; I George Lambrou; Maria Braoudaki

doi:10.1038/s41598-025-05517-9

New microRNA-based therapies reveal common targets in paediatric medulloblastoma and adult glioblastoma

Denis Mustafov, Laura Thomas, Shoib Siddiqui, I George Lambrou, Maria Braoudaki

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Abstract

Medulloblastoma (MB), the most prevalent brain malignancy in children, presents significant challenges in paediatric oncology due to its aggressiveness and potential for relapse. Tailored treatments are crucial to mitigate treatment-related toxicities and long-term side effects on developing brains. Our study aimed to identify therapeutic targets for paediatric MB and explore common miRNA biosignatures with glioblastoma (GB), the most aggressive adult brain tumour. High-throughput small-RNA sequencing identified miR-206 and miR-383 as highly downregulated in MB samples (n=9), suggesting their tumour suppressor properties. Bioinformatics analysis identified CORO1C and SV2B as their targets. RT-qPCR, western blotting, and immunohistochemistry confirmed their overexpression in MB and GB. Elevated CORO1C expression was also found in adult MB and GB tissue samples. The role of both miRNAs on their target genes was validated through in vitro functional assays. Our study uncovers the potential role of miR-206/CORO1C and miR-383/SV2B axes as innovative therapeutic targets for combating aggressive paediatric and adult brain malignancies.

Original language	English
Number of pages	18
Journal	Scientific Reports
DOIs	https://doi.org/10.1038/s41598-025-05517-9
Publication status	Published - 2 Jul 2025

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© The Author(s) 2025. This article is licensed under a Creative Commons Attribution 4.0 International License. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.
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title = "New microRNA-based therapies reveal common targets in paediatric medulloblastoma and adult glioblastoma",

abstract = "Medulloblastoma (MB), the most prevalent brain malignancy in children, presents significant challenges in paediatric oncology due to its aggressiveness and potential for relapse. Tailored treatments are crucial to mitigate treatment-related toxicities and long-term side effects on developing brains. Our study aimed to identify therapeutic targets for paediatric MB and explore common miRNA biosignatures with glioblastoma (GB), the most aggressive adult brain tumour. High-throughput small-RNA sequencing identified miR-206 and miR-383 as highly downregulated in MB samples (n=9), suggesting their tumour suppressor properties. Bioinformatics analysis identified CORO1C and SV2B as their targets. RT-qPCR, western blotting, and immunohistochemistry confirmed their overexpression in MB and GB. Elevated CORO1C expression was also found in adult MB and GB tissue samples. The role of both miRNAs on their target genes was validated through in vitro functional assays. Our study uncovers the potential role of miR-206/CORO1C and miR-383/SV2B axes as innovative therapeutic targets for combating aggressive paediatric and adult brain malignancies.",

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AU - Mustafov, Denis

AU - Thomas, Laura

AU - Siddiqui, Shoib

AU - George Lambrou, I

AU - Braoudaki, Maria

N1 - © The Author(s) 2025. This article is licensed under a Creative Commons Attribution 4.0 International License. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.

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N2 - Medulloblastoma (MB), the most prevalent brain malignancy in children, presents significant challenges in paediatric oncology due to its aggressiveness and potential for relapse. Tailored treatments are crucial to mitigate treatment-related toxicities and long-term side effects on developing brains. Our study aimed to identify therapeutic targets for paediatric MB and explore common miRNA biosignatures with glioblastoma (GB), the most aggressive adult brain tumour. High-throughput small-RNA sequencing identified miR-206 and miR-383 as highly downregulated in MB samples (n=9), suggesting their tumour suppressor properties. Bioinformatics analysis identified CORO1C and SV2B as their targets. RT-qPCR, western blotting, and immunohistochemistry confirmed their overexpression in MB and GB. Elevated CORO1C expression was also found in adult MB and GB tissue samples. The role of both miRNAs on their target genes was validated through in vitro functional assays. Our study uncovers the potential role of miR-206/CORO1C and miR-383/SV2B axes as innovative therapeutic targets for combating aggressive paediatric and adult brain malignancies.

AB - Medulloblastoma (MB), the most prevalent brain malignancy in children, presents significant challenges in paediatric oncology due to its aggressiveness and potential for relapse. Tailored treatments are crucial to mitigate treatment-related toxicities and long-term side effects on developing brains. Our study aimed to identify therapeutic targets for paediatric MB and explore common miRNA biosignatures with glioblastoma (GB), the most aggressive adult brain tumour. High-throughput small-RNA sequencing identified miR-206 and miR-383 as highly downregulated in MB samples (n=9), suggesting their tumour suppressor properties. Bioinformatics analysis identified CORO1C and SV2B as their targets. RT-qPCR, western blotting, and immunohistochemistry confirmed their overexpression in MB and GB. Elevated CORO1C expression was also found in adult MB and GB tissue samples. The role of both miRNAs on their target genes was validated through in vitro functional assays. Our study uncovers the potential role of miR-206/CORO1C and miR-383/SV2B axes as innovative therapeutic targets for combating aggressive paediatric and adult brain malignancies.

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SN - 2045-2322

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New microRNA-based therapies reveal common targets in paediatric medulloblastoma and adult glioblastoma

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