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Updated: May 29, 2025
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Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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Nucleic acid therapeutics: Past, present, and future
Sajid Naeem1,2, Ju Zhang1,2, Yang Zhang3
1College of Life Sciences and Oceanography, Shenzhen University, Shenzhen 518060, China.
Molecular Therapy. Nucleic Acids
|February 3, 2025
Summary
Nucleic acid therapeutics, including siRNA and mRNA, offer new ways to treat diseases by targeting genetic sequences. Advances in engineering and delivery are overcoming challenges for wider therapeutic use.
Area of Science:
- Biotechnology
- Molecular Biology
- Pharmacology
Background:
- Nucleic acid therapeutics target coding and non-coding sequences.
- Approved modalities include siRNA, mRNA, aptamers, and antisense oligonucleotides.
- COVID-19 vaccines and CRISPR-Cas9 have accelerated the field.
Purpose of the Study:
- To provide an overview of nucleic acid therapeutics.
- To highlight advancements in engineering, conjugation, and delivery strategies.
- To discuss the growing role of nucleic acid therapeutics in medicine.
Main Methods:
- Review of current literature on nucleic acid therapeutics.
- Analysis of engineering, conjugation, and delivery strategies.
- Discussion of regulatory approvals and clinical applications.
Main Results:
- Several nucleic acid modalities are approved for therapeutic use.
- Significant progress has been made in overcoming delivery challenges.
- CRISPR-Cas9 represents a major advancement.
Conclusions:
- Nucleic acid therapeutics are a rapidly growing area with significant potential.
- Engineering and delivery innovations are crucial for clinical success.
- These therapies are poised to play a larger role in modern medicine.
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