Question

The genetic defect - Adenosine Deaminase (ADA) deficiency may be cured permanently by

• A. periodic infusion of genetically engineered lymphocytes having functional ADA C-DNA
• B. administering adenosine deaminase activators
• C. introducing bone marrow cells producing ADA into cells at early embryonic stages
• D. enzyme replacement therapy

Answer 1

The correct option is C

introducing bone marrow cells producing ADA into cells at early embryonic stages

ADA deficiency was the first to be treated with gene therapy. Lymphocytes from patients blood are grown in culture outside, treated and then returned to the patient. But patient requires periodic infusion as their cells are mortal.

However, if the gene isolated from bone marrow cells providing ADA is introduced into cells at early embryonic stages, it could be a permanent cure.