Question

A permanent cure for ADA deficiency could be through:

• A. Bone marrow transplantation
• B. Modified lymphocyte transfusion
• C. Gene therapy at early embryonic stages
• D. Enzyme replacement therapy

Hint:

Remember that the first clinical gene therapy was given in 1990 to a 4-year-old girl with adenosine deaminase (ADA) deficiency.
Always distinguish between therapeutic modifications (which are temporary because somatic cells have a finite lifespan) and embryonic modifications (which are permanent because the gene is integrated during early development).

Answer 1

The Correct Option is C

Step 1: ADA deficiency.
ADA deficiency causes SCID. Bone marrow transplant and enzyme replacement are partial/temporary fixes.
Step 2: Permanent cure.
Gene therapy at the early embryonic stage inserts a functional ADA gene into all somatic cells permanently.
Answer: Gene therapy at early embryonic stages