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NCERT Exemplar · Q27

Q.With respect to understanding diseases, discuss the importance of transgenic animal models.

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Transgenic animal models, especially mice, allow scientists to study human diseases in a living system, test treatments, and understand genetic causes in ways that cell cultures or healthy animals cannot.

To grasp why transgenic animal models matter for understanding diseases, start with a concrete example. Imagine a scientist wants to study cystic fibrosis, a genetic disorder that clogs the lungs with thick mucus. In a petri dish, human lung cells can be grown, but they do not cough, breathe, or develop infections the way a whole body does. A healthy mouse also will not help — it does not have the faulty gene that causes the disease. So the scientist creates a transgenic mouse: a mouse whose DNA has been deliberately altered to carry the same defective human gene. That mouse will develop symptoms similar to cystic fibrosis, giving researchers a living, breathing model to study how the disease progresses and to test potential drugs.

This is the core idea. Transgenic animals are not just curiosities; they are tools that bridge the gap between molecular biology and whole-organism medicine. By inserting, deleting, or modifying specific genes, scientists can create animal models that mimic human genetic disorders. The most common animal used is the mouse, because its genome is well understood, it breeds quickly, and its physiology shares many features with humans.

Note

The NCERT textbook specifically highlights transgenic mice as models for diseases like cancer, Alzheimer’s, and cystic fibrosis. It does not mention other animals in this context, so stick to mice when answering exam questions.

Why is this important for understanding diseases? First, it reveals cause and effect. If you insert a human gene linked to a disease into a mouse, and the mouse develops that disease, you have strong evidence that the gene is responsible. For example, researchers have created transgenic mice carrying mutated forms of the BRCA1 gene, which is associated with breast cancer in humans. These mice develop tumours, confirming the gene’s role and allowing scientists to study how the cancer starts and spreads.

Second, transgenic models are indispensable for testing treatments before they reach human trials. A drug that looks promising in a test tube might fail in a living body because of how it is absorbed, metabolised, or because it causes unexpected side effects. Transgenic mice that model a disease can be given experimental drugs, and their response — whether tumours shrink, symptoms improve, or side effects appear — provides crucial data. This saves time, money, and, most importantly, prevents harm to human patients. …

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