Question

The genetic defect-Adenosine Deaminase (ADA) deficinecy may be cured permanently by

(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

Answer 1

Correct option (a) periodic infusion of genetically engineered lymphocytes having functional ADA cDNA

Explanation:

Severe Combined Immuno Deficiertcy ( 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.