Question

First gene therapy was performed for the disease:

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

Answer 1

The correct option is B ADA deficiency

The first clinical gene therapy was performed in 1990 on a 4-year old girl for treatment of a genetic defect that left her with ADA-SCID, a severe immune system deficiency.