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Updated: Jan 17, 2026
09:04
Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
Published on: September 21, 2017
9.9K
Upgrading nucleic acid and antisense therapeutics: challenges, solutions, and future directions
Abdullah Zia1, Toshifumi Yokota1,2
1Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada.
Bioanalysis
|September 22, 2025
Summary
Antisense therapeutics offer targeted gene modulation but face delivery and toxicity challenges. Advances in nucleotide chemistry and delivery platforms are crucial for expanding their use in treating genetic and acquired diseases.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- Many disease-modifying proteins lack druggable pockets for conventional therapies.
- Nucleic acid-based antisense therapeutics provide a strategy to directly modulate gene expression.
- Approved antisense drugs exist for neuromuscular, metabolic, and neurodegenerative diseases.
Purpose of the Study:
- To review the current state of antisense modalities.
- To identify obstacles hindering broader application of antisense therapies.
- To explore innovative approaches for enhancing antisense platforms.
Main Methods:
- Review of existing literature on antisense oligonucleotides (ASOs), RNA interference (RNAi), and decoy oligodeoxynucleotides (ODNs).
- Analysis of challenges including nuclease degradation, off-target binding, toxicity, tissue penetration, and endosomal escape.
- Synthesis of advancements in nucleotide chemistry, conjugation, and delivery systems.
Main Results:
- Antisense approaches have progressed significantly, leading to approved therapies.
- Key barriers to broader application include stability, specificity, delivery, and cellular uptake.
- Personalized N-of-1 therapies show promise but present ethical and cost challenges.
Conclusions:
- Overcoming current limitations requires innovation in nucleotide chemistry, conjugation, and delivery.
- Enhanced antisense platforms can expand therapeutic potential for diverse genetic and acquired diseases.
- Continued research is vital to realize the full promise of antisense therapeutics.
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