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

The genetic defect-adenosine deaminase (ADA) deficiency may be cured permanently by
  1. Enzyme replacement therapy
  2. Periodic infusion of genetically engineered lymphocytes having functional $\mathrm{ADA} \mathrm{cDNA}$
  3. Administering adenosine deaminase activators
  4. Introducing bone marrow cells producing ADA into cells at early embryonic stages

Solution

Adenosine deaminase (ADA) deficiency is a genetic disorder that affects the immune system, leading to severe combined immunodeficiency (SCID). The permanent cure involves introducing bone marrow cells that can produce the missing enzyme (ADA) into the body. This can be done by gene therapy, where the bone marrow stem cells are modified to express ADA before being reintroduced into the patient's body, thus restoring the function of the immune system.

Asked in: NEET 2009 (Mains)

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