Gene therapy developed at St. Jude Children's Research Hospital has cured infants born with X-linked severe combined immunodeficiency (SCID-X1). The children are producing functional immune cells, including T cells, B cells and natural killer (NK) cells, for the first time. The gene therapy involved collecting patients' bone marrow, then using a virus as a vector to insert a correct copy of the IL2RG gene into the genome (DNA) of patients' blood stem cells.
- New England Journal of Medicine
- California Institute of Regenerative Medicine, National Institutes of Health, Assisi Foundation of Memphis, ALSAC