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Targeting De Novo Cholesterol Synthesis as a Therapeutic Strategy in Rhabdomyosarcoma

GSE279213 Homo sapiens Expression profiling by high throughput sequencing 8 samples Submitted 2025/10/10 Platform GPL34284
Summary
Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children, but outcomes for high-risk patients remain dismal, underscoring the critical need for novel therapeutic strategies. Our study demonstrates that RMS exhibits elevated de novo cholesterol biosynthesis. Silencing key cholesterol biosynthesis genes, such as DHCR7, disrupts cell cycle progression by inducing G2/M phase arrest. Additionally, inhibiting this pathway triggers endoplasmic reticulum (ER) stress, activates the unfolded protein response (UPR), and leads to apoptosis. These findings emphasize the essential role of de novo cholesterol synthesis in RMS and highlight a novel therapeutic strategy that targets this metabolic pathway to suppress tumor growth and promote apoptosis.
Published in
Inhibiting cholesterol synthesis halts rhabdomyosarcoma growth via ER stress and cell cycle arrest
Gizaw NY, Kolari K, Kallio P et al. · EMBO molecular medicine 2025 · PMID 41249736 · doi:10.1038/s44321-025-00336-x
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Also filed as BioProject PRJNA1170548 and SRA study SRP537338. Searching any of these in the dataset finder brings you back here.

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