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Updated: Nov 24, 2025

Exploring Sequence Space to Identify Binding Sites for Regulatory RNA-Binding Proteins
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Clinical Applications of Single-Stranded Oligonucleotides: Current Landscape of Approved and In-Development

Juergen Scharner1, Isabel Aznarez1

  • 1Stoke Therapeutics, Inc., Bedford, MA, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|December 28, 2020
PubMed
Summary

Single-stranded oligonucleotides are revolutionizing medicine, offering new treatments for genetic diseases by precisely targeting gene expression and immune pathways. This versatile therapeutic platform is expanding into novel applications and vaccines.

Keywords:
CpG oligonucleotidesRNase Hantisense oligonucleotidesgapmersplicingsteric blocking

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