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Adenosine deaminase (ADA) deficiency is a genetic disorder that severely affects the immune system. Which of the following approaches provides a permanent cure for this condition?

Options

  1. ABone marrow transplantation at any stage of life
  2. BEnzyme replacement therapy by injecting functional ADA
  3. CIntroducing the ADA gene from marrow cells into cells at early embryonic stages
  4. DPeriodic infusion of genetically engineered lymphocytes

Correct answer

C. Introducing the ADA gene from marrow cells into cells at early embryonic stages

Step-by-step solution

According to NCERT, enzyme replacement therapy and bone marrow transplantation are treatments for ADA deficiency, but they are not completely curative. Gene therapy involving the periodic infusion of genetically engineered lymphocytes is also a temporary treatment because these cells have a limited lifespan and are not immortal. A permanent cure is possible only if the gene isolated from marrow cells producing ADA is introduced into the cells at early embryonic stages. Answer: Introducing the ADA gene from marrow c

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