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A First-in-Class Therapy Targeting Transcription-Replication Conflicts Drives Synthetic Lethality in Homologous Recombination Deficient Cancers

Lim, Y. C.; Lai, S. K.; Yang, R. H.; Chua, Y. K.; Patouret, R.; Manoj, V.; Soh, C.; Lebeau, B.; Cham, N.; Meng, W.; Tey, S. K.; Yeap, S. L.; Zhang, S.; Yong, J.; Ali, Z.; Mutwil, M.; Singh, P.; Fullwood, M.; Zhong, L.; Dong, X.; Chiba, S.; Koh, C. G.; Li, H. Y.

2025-05-24 cancer biology
10.1101/2025.05.21.655390 bioRxiv
Show abstract

Hidden beneath the layer of tumor malignancy lies unchecked proliferation driven by hyperactive transcription, which fosters the formation of RNA:DNA hybrids. These structural intermediates precipitate collisions between the transcription and replication machineries. Central to mitigating this genomic threat is interleukin enhancer-binding factor 2 (ILF2), which orchestrates the recruitment of RNA:DNA helicases to resolve R-loops and, in doing so, reveals cancer cells dependency on stress-mitigating mechanisms to avert genome catastrophe. Our discovery of Molephantin and its lead derivative, NYH0002 is the outcome of delineating ILF2s function. NYH002 exerts direct binding to the ILF2 complex and disrupts RNA:DNA helicase activity to elicit genome-wide DNA breakages. Tumors with elevated cyclin E and E2F1 expression are sensitive to NYH002, while those deficient in homologous recombination repair exhibit lethality. Collectively, these findings position ILF2 as a key regulator of R-loop homeostasis and reveal a therapeutic vulnerability that NYH002 exploits to suppress tumor malignancy.

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