Question

Which one of the following diseases can be corrected by gene therapy?

• A. Sleeping sickness
• B. Measles
• C. AIDS
• D. Cystic fibrosis

Hint:

The key to this question is identifying the \textbf{genetic} disease versus the \textbf{infectious} diseases. Gene therapy is designed to fix faulty genes. Sleeping sickness, measles, and AIDS are caused by external pathogens (parasite, virus), not an inherited faulty gene from the patient's parents.

Answer 1

The Correct Option is D

Step 1: Understanding the Concept:
Gene therapy is a medical strategy for treating or preventing diseases by addressing the underlying genetic cause. It's most effective for single-gene disorders (monogenic), where a working copy of the gene can replace or supplement the defective one.

Step 2: Detailed Explanation of Options:
- Sleeping sickness: This is an infection caused by the protozoan parasite {Trypanosoma brucei}. Treatment involves anti-parasitic medications, not gene therapy.
- Measles: This is an infectious disease caused by the measles virus. Vaccination is the preventative measure.
- AIDS (Acquired Immunodeficiency Syndrome): This is an infectious disease caused by HIV. It's managed with antiretroviral drugs. Gene therapy is being explored as a potential treatment, but isn't a standard corrective approach.
- Cystic fibrosis: This is a typical example of a monogenic inherited condition. It results from mutations in the CFTR gene, leading to dysfunctional protein channels. Gene therapy aims to deliver a functional CFTR gene copy to the patient's cells, particularly in the lungs, to restore normal function. It is well-suited for gene therapy.

Step 3: Final Answer:
Cystic fibrosis is the genetic disorder among the options that can be corrected by introducing a functional gene, which is the basis of gene therapy. Therefore, option (D) is correct.