Who received the first successful human gene therapy treatment in 1990?

Ashi DeSilva received the first successful human gene therapy treatment in 1990.

DeSilva was a four-year-old patient with adenosine deaminase severe combined immunodeficiency, commonly called ADA-SCID. On 14 September 1990, doctors at the National Institutes of Health removed some of her white blood cells, inserted a functioning ADA gene using a modified retroviral vector, and returned the cells to her body.

The treatment did not permanently modify every blood-forming cell, so it required repeated infusions. It nevertheless improved immune function and became a landmark demonstration that genes could be used therapeutically in a human patient.

ADA-SCID is sometimes treated with enzyme replacement, stem-cell transplantation or more durable gene therapy approaches. Emily Whitehead was the first child treated successfully with CAR-T therapy, a different form of genetic cell engineering, so the two milestones should not be confused.