Question

What is gene therapy? Illustrate this using the example of adenosine deaminase (AD deficiency.

Hint:

Gene therapy holds promise for treating genetic disorders by directly correcting the defective genes responsible for diseases.

Answer 1

Step 1: Define Gene Therapy. - Gene therapy is a technique that involves altering or replacing defective genes in an individual’s cells to treat or prevent disease.
Step 2: Example of ADA Deficiency. - Adenosine deaminase (ADA) deficiency is a genetic disorder caused by a mutation in the gene encoding the ADA enzyme, which is critical for breaking down toxic substances in the body.
Step 3: Application of Gene Therapy for ADA Deficiency. - In gene therapy for ADA deficiency, normal ADA genes are introduced into the patient's cells, typically through viral vectors. This helps the patient’s immune system function normally by producing the missing enzyme.
Final Answer: Gene therapy for ADA deficiency involves replacing the defective ADA gene with a normal one, restoring the enzyme’s function and improving the patient's immune system.