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Identification of therapeutic drug targets through genetically manipulated mice: are we getting it right?

Klaus I Matthaei1

  • 1Stem Cell & Gene Targeting Laboratory, Structural Biology Program, The John Curtin School of Medical Research, ANU College of Medicine, Biology & Environment, The Australian National University, Canberra, ACT 0200, Australia. Klaus.Matthaei@anu.edu.au

Pharmacology & Therapeutics
|April 21, 2009
PubMed

Insights

Genetically modified mice aid disease research, but genetic background matters. Analyzing diverse backgrounds can reveal gene interactions and improve therapeutic development for complex human diseases.

Area of Science:

  • Genetics and Genomics
  • Pharmacology and Therapeutics
  • Disease Modeling

Background:

  • Genetically modified mice are crucial for identifying therapeutic targets in disease control.
  • The genetic background of these mice significantly influences observed phenotypes, potentially leading to data misinterpretation.
  • Uncontrolled genetic backgrounds can obscure the true importance of specific genes in disease states.

Purpose of the Study:

  • To highlight the influence of genetic background in genetically modified mouse models.
  • To explore the potential advantages of analyzing gene function across mixed genetic backgrounds.
  • To improve the understanding of gene function and develop precise molecular therapeutics.

Main Methods:

  • Utilizing genetically modified mice with altered specific gene functions.
  • Comparing phenotypes across different and mixed genetic backgrounds.
  • Analyzing strain-specific modifications in molecular function.

Main Results:

  • Genetic background strongly impacts the phenotype of genetically modified mice.
  • Mixed genetic backgrounds may better approximate the human genome.
  • Potential to identify modifier genes and polymorphisms influencing target gene phenotypes.

Conclusions:

  • Rigorous analysis of strain-specific modifications deepens the understanding of gene function.
  • Mixed genetic backgrounds offer advantages for identifying gene interactions.
  • This approach can lead to more selective and effective molecular therapeutic strategies.

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