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Updated: May 13, 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
Design and development of antisense drugs
1National Institute of Pharmaceutical Education and Research (NIPER), Department of Biotechnology, Sector 67, SAS. Nagar, 160 062 Punjab, India +91 172 2214 682 ; +91 172 2214 692 ; ipsita@niper.ac.in.
Expert Opinion on Drug Discovery
|March 16, 2013
Summary
Antisense oligonucleotides offer high specificity as nucleic acid medicines, reducing side effects common with small molecules. However, challenges remain in targeting specific tissues and reaching the cell nucleus for effective delivery.
Area of Science:
- Biotechnology
- Molecular Biology
- Pharmacology
Background:
- Nucleic acids are revolutionizing drug discovery, offering high specificity through Watson-Crick base pairing.
- This specificity minimizes off-target effects often seen with traditional small molecule drugs.
Purpose of the Study:
- To review antisense oligonucleotides as a class of nucleic acid medicines.
- To examine the historical development and current advancement stages of these drugs.
- To critically analyze development challenges and delivery strategies.
Main Methods:
- Literature review of antisense oligonucleotide research and development.
- Analysis of drug advancement pipelines.
- Evaluation of reported delivery methods and associated challenges.
Main Results:
- Antisense oligonucleotides demonstrate high specificity and selectivity.
- Several antisense oligonucleotide-based drugs are in various stages of clinical development.
- Significant challenges exist in achieving effective tissue targeting and nuclear delivery.
Conclusions:
- Specificity is a key advantage of antisense oligonucleotides.
- Overcoming delivery barriers, particularly tissue targeting and nuclear entry, is crucial for therapeutic success.
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