Principle of Inheritance and VariationEasyBloom L3
Question
The genetic defect - Adenosine deaminase (ADA) deficiency may be cured permanently by:
Options
A.administering adenosine deaminase activators.
B.introducing bone marrow cells producing ADA into cells at early embryonic stages.
C.enzyme replacement therapy.
D.periodic infusion of genetically engineered lymphocytes having functional ADA cDNA.
Solution
Sol.(b) [Key Concept: ADA Deficiency — Gene Therapy] ADA (adenosine deaminase) deficiency causes SCID (Severe Combined Immunodeficiency). It can be cured PERMANENTLY by introducing bone marrow cells (stem cells) producing ADA into the patient at early embryonic stages. These stem cells will multiply and produce ADA throughout life. Enzyme replacement therapy (option c) and genetically engineered lymphocytes (option d) are temporary measures requiring repeated treatment; introducing ADA-producing stem cells provides a permanent cure.
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