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Updated: Sep 20, 2025

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
Published on: September 21, 2017
Modulation of DNA transcription: The future of ASO therapeutics?
1RNA Therapeutic Institute, UMASS Chan Medical School, Worcester, MA 01581, USA.
Abstract:
In this issue of Cell, Kornblihtt and colleagues report a strategy to improve antisense oligonucleotide spinal muscular atrophy therapy. They discover that the oligonucleotide drug nusinersen, which induces exon inclusion, also promotes repressive chromatin modifications, which in turn work against exon inclusion. Notably, co-administration of histone deacetylase inhibitors counteracted this effect to augment exon inclusion.
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