RNA-mediated inhibition of mitochondrial SHMT2 impairs cancer cell proliferation

Abstract Targeting metabolic reprogramming is crucial for cancer treatment. Recent advances highlight RNA’s ability to directly regulate enzyme activity through riboregulation. In this study, we used an RNA-based approach to inhibit the mitochondrial enzyme Serine hydroxymethyltransferase 2 (SHMT2),...

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Main Authors: Francesca Romana Liberati, Sharon Spizzichino, Sara Di Russo, Giulia Elizabeth Borsatti, Agnese Riva, Maria Chiara Magnifico, Amani Bouzidi, Giorgio Giardina, Marzia Arese, Chiara Scribani Rossi, Dalila Boi, Giovanna Boumis, Federica Di Fonzo, Giulia Guarguaglini, Roberto Contestabile, Angela Tramonti, Alberto Macone, Alessandro Paiardini, Serena Rinaldo, Alessio Paone, Francesca Cutruzzolà
Format: Article
Language:English
Published: Nature Publishing Group 2025-08-01
Series:Cell Death Discovery
Online Access:https://doi.org/10.1038/s41420-025-02646-y
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Summary:Abstract Targeting metabolic reprogramming is crucial for cancer treatment. Recent advances highlight RNA’s ability to directly regulate enzyme activity through riboregulation. In this study, we used an RNA-based approach to inhibit the mitochondrial enzyme Serine hydroxymethyltransferase 2 (SHMT2), which lacks a selective in vivo inhibitor. SHMT2, often overexpressed in various cancers, is pivotal in one-carbon metabolism, a pathway vital for cell proliferation. Our results show that RNA effectively inhibits SHMT2’s serine-to-glycine conversion in vitro (IC50 = 4.4 ± 0.2 nM). By using a mitochondrial import signal, we successfully delivered the inhibitory RNA into the mitochondria of lung cancer cells, reducing cell viability in vitro and tumor growth in vivo in a xenograft mouse model. These findings suggest that RNA-based strategies could be extended to selectively target other RNA-binding metabolic enzymes, offering potential solutions where small molecule inhibitors fall short or to counteract drug resistance.
ISSN:2058-7716