Germ Cell Modifications (Gene Therapy), Ethics of

The concept of gene therapy first appeared in 1972. But it was in 1990 that the first gene therapy experiment ever conducted in the United States was in fact approved by the Food and Drug Administration (FDA). At that time, the patient was a four-year-old girl named Ashanti DeSilva, born with an autosomal recessive metabolic disorder displaying a missing enzyme called adenosine deaminase (ADA). Individuals with ADA deficiency usually have a compromised immune system.

W. French Anderson of the National Heart, Lung, and Blood Institute and his team collected blood from Ashanti and replaced the defective gene with what they called a “functional variant.” The therapy succeeded in partially restoring Ashanti’s immunity, partially, because it stimulated production of the missing enzyme but never generated new cells ...