(Photo: Key City).
Researchers from the Leiden University Medical Center have successfully treated a baby with the serious congenital immune disease SCID with stem cell gene therapy. It is the first stem cell gene therapy from Dutch soil that has been administered to a patient. Worldwide, it is the first stem cell gene therapy for this form of SCID. The disease has been cured thanks to this therapy and the baby is doing well.
The treated patient is a baby just a few months old with the immune disease Severe Combined Immunodeficiency (SCID). Due to a DNA error in the blood stem cells, no immune cells are produced and the body cannot defend itself against infections. The developed stem cell gene therapy can correct this DNA error.
“The repaired stem cells have subsequently grown into a well-functioning immune system in the patient’s body,” says Arjan Lankester, professor of Pediatrics and Stem Cell Transplantation. “The baby has passed the treatment without any problems and the immune system is responding well to the usual vaccinations for newborns. A fantastic result.”
Leiden Society