NEET2009ZoologyPrinciples of Inheritance and VariationActual
The genetic defect-Adenosine Deaminase (ADA) deficiency may be cured permanentlyby
Options
- Aperiodic infusion of genetically engineered lymphocytes having functional ADA cDNA
- Badministering adenosine deaminase activators
- Cintroducing bone marrow cells producing ADA into cells at early embryonic stages
- Denzyme replacement therapy
Correct answer
C. introducing bone marrow cells producing ADA into cells at early embryonic stages
Step-by-step 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.