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
Correct Answer: A
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.
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