First approved gene therapy trial

First approved gene therapy trial
Who
NIH Clinical Centre
What
First
Where
United States
When
14 September 1990

On 14 September 1990, a four-year-old girl participated in the world's first approved gene therapy trial, at the National Institutes of Health Clinical Center, Maryland, USA. She was born with a rare genetic disorder called adenosine deaminase (ADA) deficiency, a disease that left her immune system defenceless against infections. During the procedure, white blood cells were taken and the normal genes for making adenosine deaminase were inserted into them. The corrected cells were then re-injected back into the girl, who she showed good signs of recovery, not least in the reduction of the huge number of coughs and colds she had previously endured.