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Gene targeted agents: new opportunities for rational drug development
1Nuclear Medicine Department, National Institutes of Health, Bethesda, MD 20892, USA. winters@nmdhst.cc.niih.gov
Current Opinion in Molecular Therapeutics
|March 16, 2001
Summary
Sequence-specific DNA-binding ligands offer novel therapeutic strategies beyond traditional drug development. Triple-helix forming oligonucleotides show significant promise for regulating gene expression and structure.
Area of Science:
- Molecular Biology
- Genetics
- Drug Discovery
Background:
- Traditional drug development targets protein activity.
- DNA sequence knowledge enables rational design of DNA-level therapeutics.
- Oligonucleotides, peptide nucleic acids, and polyamides are key DNA-binding ligands.
Purpose of the Study:
- To review recent advances in sequence-specific DNA-binding ligands for modifying gene expression and structure.
- To highlight the therapeutic potential of these novel agents.
- To emphasize the role of triplex-forming oligonucleotides.
Main Methods:
- Investigation of various DNA-binding ligands (oligonucleotides, PNA, polyamides).
- Exploration of strategies for transient or permanent gene expression alteration.
- Focus on recent therapeutic applications and advancements.
Main Results:
- Demonstrated therapeutic potential of DNA-binding ligands in the last 2-3 years.
- Triple-helix forming oligonucleotides show particular efficacy.
- Applications include transcriptional regulation and site-specific mutations.
Conclusions:
- Sequence-specific DNA-binding ligands represent a promising frontier in therapeutic development.
- Triple-helix forming oligonucleotides are effective agents for gene modulation.
- These ligands offer precise control over gene expression and DNA structure.