Q181 / 200 · NEET 2009 (Screening) · Zoology

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

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

Solution

Severe combined immunodeficiency (SCID) caused by adenosine deaminase deficiency (ADA) is the first genetic disorder to be treated with gene therapy. T-cell-directed gene transfer was useful in the treatment of ADA-SCID, whereas the retroviral-mediated gene transfer to haematopoietic stem cells was insufficient for achievement of clinical benefits.

Hence, the correct answer is option: 'C'.

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