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Published on: November 8, 2014
Mouse genetics in drug target discovery and validation: no simple answers to complex problems
1University of Oxford, Genetics Unit, Department of Biochemistry, South Parks Road, Oxford OX1 3QU, UK +44 1865 275248 ; +44 1865 275259 ; andreas.russ@bioch.ox.ac.uk.
Expert Opinion on Drug Discovery
|March 15, 2013
Summary
Mouse genetics is crucial for early drug discovery, validating therapeutic targets before pharmacological data is available. Unexpected findings in model organisms offer vital insights into complex diseases like obesity and diabetes.
Area of Science:
- Genetics
- Pharmacology
- Drug Discovery
Background:
- Early-stage drug discovery requires experimental validation of therapeutic targets.
- Genetic analysis in model organisms is essential when pharmacological data is scarce.
- Mouse genetics plays a pivotal role in the initial phases of drug development.
Purpose of the Study:
- To discuss the potential and pitfalls of using mouse genetics in drug discovery.
- To highlight the importance of unexpected genetic findings in understanding disease.
- To illustrate these concepts with examples from obesity and diabetes research.
Main Methods:
- Review of current literature on mouse genetics in drug discovery.
- Analysis of case studies from obesity and diabetes research.
- Discussion of concepts such as endophenotypes, functional challenges, and developmental compensation.
Main Results:
- Mouse genetics provides valuable insights into drug target validation.
- Unexpected genetic results can reveal complex biological mechanisms.
- Endophenotypes, functional challenges, and developmental compensation influence outcomes.
- Findings from mouse models are critical for refining pathophysiological models.
Conclusions:
- Mouse genetics is an indispensable tool in early drug discovery.
- Seemingly problematic results from mouse models offer crucial biological information.
- Understanding these complexities improves our models of human diseases like obesity and diabetes.
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