Human Health and DiseaseHard
Question
The genetic defect-Adenosine Deaminase (ADA) deficiency may by cured permanently by
Options
A.Periodic infusion of genetically engineered lymphocytes having functional ADA cDNA
B.administering adenosine deaminase activators
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 trans fer 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.
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