Article

Small activating RNA AW1-51 (CEBPA-51) elicits targeted DNA demethylation to promote gene activation

2025-09-30

Abstract excerpt

Small activating RNAs are short double-stranded RNAs designed to upregulate transcription of target genes. By this virtue, they can be used to restore expression of genes frequently silenced in cancer. AW1-51 (also referred to as CEBPA-51), the first small activating RNA therapeutic to enter clinical evaluation, has demonstrated biological activity and safety in Phase II trials for hepatocellular carcinoma, both a...

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Literature Corpus work: 771a81f1-9131-5003-9fd4-53241103bcd8
DOI: 10.1101/2025.09.29.679274

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