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Overview of target validation and the impact of oligonucleotides
Simon W Jones1, Mark A Lindsay
1AstraZeneca Pharmaceuticals, Alderley Park, Macclesfield, Cheshire, SK10 4TG, UK.
Abstract:
During target validation, researchers attempt to modulate the activity of potential drug targets in relevant cell and/or animal disease models in order to identify those that might be expected to have therapeutic benefit in human patients. This has become increasingly important with the large expansion of potential targets identified by the human genome project and as a result of the spiralling costs of bringing drugs to market. This review will present an overview of the target-validation mechanism and examine the strengths and weaknesses of using oligonucleotide-based technologies such as antisense, short interfering RNA and aptamers.
Insights
Target validation is crucial for drug discovery, especially with the human genome project
Area of Science:
- Drug discovery and development
- Genomics
- Molecular biology
Background:
- The human genome project has identified numerous potential drug targets.
- Rising drug development costs necessitate efficient target validation.
- Modulating target activity in disease models is key for therapeutic assessment.
Purpose of the Study:
- To provide an overview of target validation mechanisms.
- To analyze oligonucleotide-based technologies for target validation.
- To discuss the strengths and weaknesses of antisense, siRNA, and aptamer technologies.
Main Methods:
- Review of target validation strategies.
- Examination of oligonucleotide-based therapeutic technologies.
- Analysis of antisense, short interfering RNA (siRNA), and aptamer applications.
Main Results:
- Oligonucleotide technologies offer diverse approaches to target modulation.
- Each technology presents unique advantages and limitations in target validation.
- Effective target validation is essential for successful drug development.
Conclusions:
- Oligonucleotide-based tools are vital for validating drug targets.
- Understanding the strengths and weaknesses of these technologies aids in selection.
- Efficient target validation accelerates the development of therapeutic agents.
