NEET Biology question

The genetic defect adenosine deaminase (ADA) deficiency may be cured permanently by

(A)Administering adenosine deaminase activators
(B)Introducing bone-marrow cells producing ADA into cells at early embryonic stagesCorrect
(C)Enzyme replacement therapy
(D)Periodic infusion of genetically engineered lymphocytes having functional ADA cDNA

Answer: (B)

Solution

ADA deficiency can be permanently cured only by introducing a functional ADA gene into cells at an early embryonic stage (or into haematopoietic stem cells that will give rise to lymphocytes throughout life), so the corrected cells (and their descendants) continue producing the enzyme for the person's lifetime.

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Question ID VK-120235