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Utilization of genetically altered animals in the pharmaceutical industry.

D G Rudmann1, S K Durham

  • 1Department of Pathology, DuPont Pharmaceuticals Company, Newark, Delaware 19714, USA. daniel.g.rudmann.2@dupontpharma.com

Toxicologic Pathology
|June 15, 1999
PubMed
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Genetically altered mice, including knockout models, are crucial tools in pharmaceutical research for understanding gene function and modeling human diseases. These models accelerate drug discovery and development by aiding in risk assessment and toxicity evaluations.

Area of Science:

  • Pharmacology and Toxicology
  • Genetics and Genomics
  • Biomedical Research

Background:

  • Genetically modified mice, such as transgenic and knockout models, are instrumental in elucidating gene product functions in vivo.
  • These models are widely adopted in the pharmaceutical industry for drug discovery and the development of disease models.

Purpose of the Study:

  • To highlight the utility of genetically altered mice in pharmaceutical research, focusing on drug discovery and development.
  • To examine the role of the NF-kappaB gene family in immune and inflammatory responses using genetically modified mice.
  • To discuss the application of these models in evaluating drug safety, including carcinogenesis and toxicity.

Main Methods:

  • Utilizing transgenic and gene-deleted (knockout) mouse models to study gene function and interactions.

Related Experiment Videos

  • Employing genetically altered animals to investigate the NF-kappaB family in immune and inflammatory pathways.
  • Applying these models for risk assessment, carcinogenicity, xenobiotic metabolism, and toxicity studies.

    • Main Results:

    • Genetically altered mice facilitate the characterization of gene product functions and the establishment of disease models.
    • Studies involving the NF-kappaB family in genetically modified mice revealed their critical role in early immune and inflammatory responses.
    • The p53 knockout mouse model demonstrates potential to enhance the sensitivity and reduce the cost of carcinogenicity bioassays.

    Conclusions:

    • Genetically altered mice are powerful tools in pharmaceutical research, advancing drug discovery and development.
    • Careful interpretation of data from genetically altered mice is essential, considering potential compensatory mechanisms and genetic background effects.
    • Despite limitations, these models significantly contribute to understanding biological processes and evaluating drug candidates.