Question

The first instance of gene therapy was done for:

• A. Alkaptonuria
• B. ADA deficiency
• C. Down’s syndrome
• D. Cystic fibrosis

Answer 1

The correct option is B ADA deficiency

the first instance of gene therapy was done in 1990 to aid a 4 year old girl with ADA deficiency. ADA enzyme is essential for proper functioning of immune system. By complementing the gene of ADA enzyme and introducing them into the donor cells of the girl, her body was able to supplement the deficient ADA enzyme. Something that would not have been possible with over the counter drugs.