Prev : Next The first gene therapy
Restoring a life
Dr. W. French Anderson of the National Heart, Lung, and Blood Institute presides over the first experimental use of a gene therapy. The patient is a four-year old girl name Ashanti DeSilva, who was born with an autosomal recessive metabolic disorder that manifests as a lack of the enzyme adenosine deaminase (ADA). The root cause is a single defective gene. Patients with ADA deficiency suffer from severe immunodeficiency. In the absence of the enzyme, substances toxic to lymphocytes accumulate and immune function is either severely compromised or completely absent. Dr. Anderson’s team drew blood from their patient, and replaced the defective gene with a functional variant. The therapy partially restores Ashanti’s immune system. It temporarily stimulates production of the missing enzyme, but does not generate new cells with functional genes. Ashanti continues to receive injections of corrected T-cells every two months. She is able to lead a normal life.