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In pursuit of antisense
1Gilead Sciences Inc., Foster City, California 94404, USA.
Nature
|November 7, 1996
Summary
First-generation antisense oligodeoxynucleotides (ODNs) show promise in clinical trials. Preclinical studies indicate enhanced tissue permeability for phosphorothioate ODNs, suggesting potential for pharmacological applications.
Area of Science:
- Pharmacology
- Molecular Biology
- Biochemistry
Background:
- First-generation antisense oligodeoxynucleotides (ODNs) are in clinical trials.
- Their therapeutic effects might stem from mechanisms beyond RNA binding.
- Understanding ODN behavior in biological systems is crucial.
Purpose of the Study:
- To evaluate the pharmacological potential of antisense oligodeoxynucleotides (ODNs).
- To investigate the tissue permeability of phosphorothioate ODNs in preclinical models.
Main Methods:
- Analysis of preclinical animal studies on ODN tissue distribution.
- Comparison of ODN permeability in animal tissues versus cell culture.
Main Results:
- Phosphorothioate ODNs demonstrate greater permeability in certain animal tissues than previously observed in cell cultures.
- This suggests improved bioavailability and potential for targeted delivery.
Conclusions:
- Antisense mechanisms mediated by ODNs may be pharmacologically viable.
- Enhanced tissue permeability of phosphorothioate ODNs supports their therapeutic potential.