First Human Gene Therapy (ADA-SCID)
In September 1990 doctors at the US National Institutes of Health gave a four-year-old girl with a rare immune disease her own white blood cells carrying a working copy of her faulty gene.
Open in the interactive tree →Under W. French Anderson, white blood cells of Ashanthi DeSilva, who had the immune disease ADA-SCID, were taken out of her body, given a healthy ADA gene with a retrovirus and infused back; she also kept getting enzyme injections, and the effect faded. After the death of Jesse Gelsinger in a 1999 trial and leukemia in some children treated for SCID in the early 2000s, researchers built safer vectors. The take-out, change and return method is now used for Strimvelis (approved May 2016) and for CAR-T cells, and gene delivery in the body led to Luxturna (December 2017).
Prerequisites
- Cell Culture and HeLa Cells1951White blood cells were grown and modified in the lab before being returned.
- Reverse Transcriptase (RNA to DNA)1970A retrovirus, a virus that inserts its genes into cells, carried the gene in.
- Genetic Engineering1973A working copy of the faulty gene had to be cloned first.
Unlocks
- CAR-T Cell Therapy2017CAR-T cells are a patient's own cells changed outside the body and returned.
- Gene Therapy (AAV Vectors)2017AAV products continue the idea of delivering a working gene.
- First CRISPR Medicines2023Edited blood stem cells are returned to the patient, as in gene therapy.
Sources
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