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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
Recent progress in oligonucleotide therapeutics: antisense to aptamers
1The University of Toledo, Department of Biological Sciences, Toledo, OH 43606, USA +1 419 530 1555 ; +1 419 530 7737 ; dleaman@utnet.utoledo.edu.
Expert Opinion on Drug Discovery
|March 20, 2013
Summary
Antisense technology shows promise for treating diseases by targeting RNA. Advances in stabilization and delivery offer new hope for nucleic acid therapeutics despite ongoing technical challenges.
Area of Science:
- Biotechnology
- Molecular Biology
- Therapeutics
Background:
- RNA-targeting strategies, including antisense technology, hold significant potential for treating diverse human diseases.
- Despite methodological advancements in RNA degradation over the last decade, persistent technological hurdles challenge nucleic acid-based drugs.
Purpose of the Study:
- To present an updated overview of the clinical progression of various antisense compounds.
- To explore strategies aimed at enhancing the efficacy of antisense compounds and other nucleic acid therapeutics.
Main Methods:
- Review of clinical advancements in antisense therapeutics.
- Discussion of sugar or backbone modifications to improve antisense efficacy.
- Exploration of delivery approaches for enhanced therapeutic outcomes.
- Consideration of alternative nucleic acid-based therapeutic strategies.
Main Results:
- Highlighting key clinical advances in antisense drug development.
- Detailing modifications (sugar/backbone) and delivery methods that improve antisense compound efficacy.
- Presenting alternative nucleic acid strategies for therapeutic applications.
Conclusions:
- Technological challenges continue to impede the clinical advancement of antisense therapies.
- Recent progress in stabilization and delivery techniques offers renewed optimism for the clinical application of nucleic acids as pharmaceuticals.
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