The genetic defect-Adenosine Deaminase (ADA) deficiency may be cured permanently by
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periodic infusion of genetically engineered lymphocytes having functional ADA cDNA
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administering denosine deaminase activators
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introducing bone marrow cells producing ADA into cells at early embryonic stages
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enzyme replacement therapy
A.
periodic infusion of genetically engineered lymphocytes having functional ADA cDNA
severe combined immunodeficiency (SCID) caused by adenosine deaminase deficiency (AIDA) 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 the achievement of clinical benefits.
