Question

The human protein named $\alpha$-1-antitrypsin, obtained from transgenic animals, is used for the treatment of ________.

• A. Alzheimer's disease
• B. Emphysema
• C. Cystic fibrosis
• D. Rheumatoid arthritis

Hint:

Logic Tip: Connect the "anti-trypsin" (an enzyme inhibitor) directly to lung protection. Emphysema is a disease where lung tissue is actively digested. Supplying the inhibitor stops the destruction!

Answer 1

The Correct Option is B

Concept:
Transgenic animals are animals whose DNA has been manipulated to possess and express an extra (foreign) gene. One major application of transgenic animals is the production of valuable biological products, particularly human proteins used to treat specific genetic or metabolic diseases.
Step 1:
The protein $\alpha$-1-antitrypsin (AAT) is a protease inhibitor normally produced by the liver. Its primary role in the human body is to protect the lungs from neutrophil elastase, an enzyme that breaks down elastin in alveolar walls during inflammation.
Step 2:
A genetic deficiency in $\alpha$-1-antitrypsin leads to unchecked elastase activity in the lungs. This chronic tissue degradation breaks down the delicate alveolar walls, drastically reducing the surface area for gas exchange, a condition clinically diagnosed as Emphysema.
Step 3:
To treat patients suffering from this specific form of emphysema, researchers developed transgenic animals (such as transgenic sheep) that contain the human gene for $\alpha$-1-antitrypsin. These animals secrete large quantities of the functional human protein into their milk, which is then harvested, purified, and administered to patients.
Step 4:
Alzheimer's disease, Cystic fibrosis, and Rheumatoid arthritis have different underlying pathophysiologies and are not currently treated using the $\alpha$-1-antitrypsin protein produced by transgenic animals.
Step 5:
Therefore, $\alpha$-1-antitrypsin is explicitly produced and used for the treatment of Emphysema.