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 approach that treats or prevents disease by correcting the underlying genetic problem. It is most directly applicable to diseases caused by a single faulty gene (monogenic disorders), where a functional copy of the gene can be introduced to replace or supplement the defective one.

Step 2: Detailed Explanation of Options:
- Sleeping sickness: This is an infectious disease caused by the protozoan parasite {Trypanosoma brucei}. It is treated with anti-parasitic drugs, not gene therapy.
- Measles: This is an infectious disease caused by the measles virus. It is preventable by vaccination.
- AIDS (Acquired Immunodeficiency Syndrome): This is an infectious disease caused by the Human Immunodeficiency Virus (HIV). It is managed with antiretroviral drugs. While gene therapy is being researched as a potential treatment for HIV, it is not a standard correction method.
- Cystic fibrosis: This is a classic example of a monogenic inherited disease. It is caused by mutations in the CFTR gene, which leads to the production of faulty protein channels. Gene therapy aims to deliver a correct copy of the CFTR gene to the cells of the patient, particularly in the lungs, to restore normal function. It is a prime candidate for correction by gene therapy.

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