Britannica
Sources used in this answer
How do genetically modified animal models help scientists study human genetic diseases?
Created from Britannica articles
using AI.
AI can make mistakes, so verify using Britannica articles.
genetic engineering
The generation of vaccines relies heavily on techniques in genetic engineering.
Genetically modified (GM) animal models are essential tools in biomedical research, allowing scientists to investigate the pathology of human genetic diseases and develop novel therapies. Because humans and many laboratory animals, such as mice, share a significant number of genes, these models provide critical insights into gene function and the biological roles of genes involved in disease.1234
Scientists use several approaches to create these models:
- Knockout Models: By inactivating or "knocking out" a specific gene, researchers can observe changes in phenotype to understand the gene's normal function and its contribution to conditions like cancer, cystic fibrosis, and Parkinson disease.34
- Transgenic Models: Researchers can insert specific genetic material—such as mutations found in human patients—into an animal's genome to replicate the disease state, enabling the testing of new drug therapies.24
- Gene Editing: Technologies like CRISPR-Cas9 allow for precise modifications, such as correcting genetic errors in animal models to achieve clinical rescue.5
While these models are invaluable, ethical principles dictate that research must balance scientific progress with animal welfare, adhering to the "three Rs": replacement, reduction, and refinement.4