The first clinical gene therapy was given for the treatment of which one of the following diseases?
Answer & explanation
Correct answer: option 4
The correct answer is Option (4) → Adenosine deaminase (ADA) deficiency
Gene therapy corrects a gene defect by delivering a normal gene into the person’s cells. The first clinical gene therapy was given in 1990 to a 4-year old girl with adenosine deaminase (ADA) deficiency. In this disorder the gene for the enzyme ADA is deleted, and the enzyme is needed for the immune system to work. Lymphocytes from her blood were grown outside the body, a functional ADA cDNA was put into them with a retroviral vector, and the cells were returned to her.
NCERT Extract:
Gene Therapy – “… The first clinical gene therapy was given in 1990 to a 4-year old girl with adenosine deaminase (ADA) deficiency. This enzyme is crucial for the immune system to function. The disorder is caused due to the deletion of the gene for adenosine deaminase.”