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Updated: May 21, 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
Making sense of therapeutics using antisense technology.
1National Institute of Pharmaceutical Education and Research (NIPER), Department of Biotechnology , Sector 67, S.A.S. Nagar, Punjab 160 062 , India ipsita@niper.ac.in.
Expert Opinion on Drug Discovery
|June 1, 2012
Summary
Antisense oligonucleotides (ASOs) offer targeted mRNA destruction for genetic disease correction. Key challenges remain in ASO stability, delivery, and bioavailability for effective therapeutic applications.
Area of Science:
- Molecular Biology
- Genetics
- Pharmacology
Background:
- Antisense oligonucleotides (ASOs) are synthetic DNA sequences targeting specific mRNA molecules.
- ASOs induce mRNA cleavage, leading to targeted gene silencing and potential disease correction.
- The therapeutic potential of ASOs is underscored by their diverse applications in genetic aberration treatment.
Purpose of the Study:
- This review examines the therapeutic applications of ASOs.
- It details ASO drugs in preclinical and clinical trials.
- The review also addresses challenges and strategies in ASO development and delivery.
Main Methods:
- Literature review of ASO therapeutic effects.
- Discussion of ASO drugs in various trial stages.
- Analysis of ASO delivery and stabilization strategies.
Main Results:
- ASO design for mRNA targeting is established.
- Numerous ASO applications demonstrate therapeutic promise.
- Strategies for ASO stabilization and delivery are crucial for efficacy.
Conclusions:
- ASO technology shows significant advances for therapeutic applications.
- In vivo stability, targeted delivery, and bioavailability are critical hurdles.
- Overcoming these challenges is key to realizing the full potential of ASO therapeutics.
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