Q. The first clinical gene therapy was done for treatment of
Solution:
Solution:
The first clinical gene therapy trial was given in the year, 1990, to a four-year-old girl with adenosine deaminase (ADA) deficiency. Adenosine deaminase (ADA) deficiency can lead to severe combined immunodeficiency (SCID). In gene therapy, the cells are extracted from the bone marrow of the patient, cultured and the functional ADA/cDNA is then transfected through adenoviral vector. SCID is a rare genetic condition, an autosomal recessive disorder. The basic characteristic of this disease is that the infants lack the two primary lymphocytes B and T lymphocytes, which form an integral part of the adaptive immune response. Hence, the affected individual will be immunologically compromised, becoming prime targets for numerous life-threatening infections.