Article

A chemoinformatics-guided platform for efficient discovery of RNA-binding small molecules: Proof-of-concept for myotonic dystrophy type 1

2026-05-13

Abstract excerpt

Structured RNAs cause human diseases but remain challenging to target selectively with small molecules. Here, we report a chemoinformatics-guided discovery framework that integrates fingerprint-based molecular design, experimental validation, and mechanistic profiling to identify small molecules that bind highly structured, disease-associated RNAs. Using an RNA-binder fingerprint derived from known ligands, a Tver...

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Literature Corpus work: 75b7df3c-07c3-599e-aabe-f7a0a7dcb035
DOI: 10.64898/2026.05.08.723748

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