Research reveals that RNA boosts condensate nucleation and enhances transcription in ENL mutant cells, suggesting a new target for cancer therapy.
Aberrant chromatin-associated condensates have emerged as drivers of transcriptional dysregulation in cancer, yet the mechanisms regulating their formation and function remain poorly understood. Gain-of-function mutations in the histone acetylation reader ENL, found in leukemia and Wilms tumor, drive oncogenesis by inducing condensate formation at highly selective genomic loci. Here, we uncover a critical role for RNA in reinforcing the nucleation, chromatin engagement, and oncogenic activity of ENL mutant condensates. Mutant ENL binds RNA via a conserved basic patch within its YEATS domain, and this interaction enhances condensate formation both in vitro and across diverse cellular contexts. Using a chemically inducible condensate displacement and re-nucleation system, we show that blocking ENL- RNA interactions or transcription impairs condensate reformation at endogenous targets. RNA interactions preferentially enhance ENL occupancy at condensate-permissive loci, leading to increased transcriptional bursting and robust gene activation at the single-cell and single-allele level. In mouse models, disrupting ENL-RNA interactions diminishes condensate formation and oncogenic transcription in hematopoietic stem and progenitor cells, thereby suppressing ENL mutation-driven leukemogenesis. Together, these findings establish RNA as an active cofactor that pathogenic condensates exploit to reinforce locus-specific nucleation and amplify transcriptional programs that drive disease.
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Budinich et al. (2025) studied this question.
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