The first gene therapy (somatic enzyme) was successfully done in: (A) SCID, (B) Phenylketonuria, (C) Thalassemia, (D) Cystic fibrosis
Answer is A. Deficiency of adenosine deaminase, a condition that affects lymphocytes paicularly, was the first disease to be treated by gene therapy. Deficiency of adenosine deaminase accounts for 15% of all cases of severe combined immunodeficiency (SCID)In 1990, Dr. William French Anderson introduced a new copy the gene, carried on a retroviral vector, into a 4-year-old girl suffering from severe combined immunodeficiency (Bubble boy disease).Ref: Harper 30th edition pg: 699