Assertion: The first clinical gene therapy for ADA was given to cure SCID.
Reason: The normal gene was delivered into the patient's cells using a retroviral vector.
ADA deficiency causes an immune disorder known as SCID (Severe Combined Immuno-Deficiency disease). In this condition patients contain mutated ADA genes, thus ADA enzyme becomes deficient. Functional ADA gene is inserted into the patient's cell to treat this disease. Such a process of introducing normal functional genes into cells, which contains the defective gene correcting a genetic disorder is called gene therapy.
First gene therapy was done to cure SCID and the normal gene was introduced using a retroviral vector. The first approved gene therapy in the US took place on 14 September 1990, at the National Institutes of Health (NIH) under the direction of William French Anderson. A four-year old girl named Ashanthi De Silva was given the first clinical gene therapy to treat ADA deficiency.