Related Experiment Video

Updated: Aug 6, 2026

A Genetically Engineered Mouse Model of Sporadic Colorectal Cancer
06:01

A Genetically Engineered Mouse Model of Sporadic Colorectal Cancer

Published on: July 6, 2017

The mighty mouse: genetically engineered mouse models in cancer drug development

Norman E Sharpless1, Ronald A Depinho

  • 1Departments of Medicine and Genetics, The Lineberger Comprehensive Cancer Center, The University of North Carolina, Chapel Hill, North Carolina 27599-7295, USA. nes@med.unc.edu

Nature Reviews. Drug Discovery
|August 18, 2006
PubMed

Insights

Standard preclinical cancer drug testing models like xenografts have limitations. Novel genetically engineered mouse models offer better human cancer mimicry, potentially improving drug development success rates and clinical translation.

Area of Science:

  • Oncology
  • Translational Medicine
  • Preclinical Drug Development

Background:

  • Standard preclinical models, such as xenograft mouse models, face significant limitations in accurately predicting human cancer drug efficacy.
  • These limitations hinder the translation of basic cancer research discoveries into effective clinical treatments.

Purpose of the Study:

  • To discuss the established uses and limitations of xenograft mouse models in cancer drug development.
  • To explore the potential of novel genetically engineered mouse models (GEMMs) that better recapitulate human cancer evolution.
  • To identify opportunities and challenges in applying GEMMs for improved cancer drug discovery.

Main Methods:

  • Review of existing literature on xenograft models for cancer drug evaluation.
  • Description of the characteristics and advantages of genetically engineered mouse models.
  • Analysis of the application of GEMMs in key stages of drug development.

Main Results:

  • Xenograft models have inherent limitations in mimicking human cancer's genetic and biological complexity.
  • Genetically engineered mouse models offer a more faithful representation of human cancer progression and heterogeneity.
  • GEMMs show promise in target validation, response assessment, pharmacodynamic studies, resistance modeling, and toxicity evaluation.

Conclusions:

  • Greater utilization of genetically engineered mouse models can significantly enhance the success rate of anticancer drug development.
  • Addressing the challenges associated with GEMMs is crucial for their widespread adoption.
  • Improved preclinical models are essential for bridging the gap between basic research and clinical benefit in oncology.

More Related Videos

Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor07:21

Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor

Published on: May 2, 2022

Generation of Tumor Organoids from Genetically Engineered Mouse Models of Prostate Cancer08:54

Generation of Tumor Organoids from Genetically Engineered Mouse Models of Prostate Cancer

Published on: June 13, 2019

Related Concept Videos

Mouse Models of Cancer Study02:43

Mouse Models of Cancer Study

Mice have long served as models for studying human biology and pathology because of their phylogenetic and physiological similarity with humans. They are also easy to maintain and breed in the laboratory, and hence, many inbred strains are now available for research. Studies on mice have contributed immeasurably to our understanding of cancer biology.
The development of transgenic, knockout, and knock-in mice has led to an exponential increase in their use as model organisms in research,...
Mouse Models of Cancer Study02:43

Mouse Models of Cancer Study

Mice have long served as models for studying human biology and pathology because of their phylogenetic and physiological similarity with humans. They are also easy to maintain and breed in the laboratory, and hence, many inbred strains are now available for research. Studies on mice have contributed immeasurably to our understanding of cancer biology.
The development of transgenic, knockout, and knock-in mice has led to an exponential increase in their use as model organisms in research,...
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.