The genetic defect-Adenosine Deaminase (ADA) deficiency may be cured permanentlyby
The genetic defect-Adenosine Deaminase (ADA) deficiency may be cured permanentlyby
periodic infusion of genetically engineered lymphocytes having functional ADA cDNA
administering adenosine deaminase activators
introducing bone marrow cells producing ADA into cells at early embryonic stages
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.