New gene therapy from St. Jude Children’s Research Hospital cures infants with severe immunodeficiency

May 10, 2019

A newly developed genetic therapy created at St. Jude Children’s Research Hospital has helped cure newborns with X-linked immunodeficiency. As published in the New England Journal of Medicine, the children are now able to produce properly functioning immune cells, such as B cells, T cells and natural killer cells.

Lead author of the study, Ewelina Mamcarz, M.D., states, “the patients are now toddlers, able to respond to vaccinations and create immune cells to defend against infections as they move on through life.” These children suffer from SCID-X1, a common form of SCID, which is a genetic disorder characterized by little to no immune system.

In this clinical trial, 10 infants had received the therapy. After just three months of treatment, immune cells were found to be present within the blood of 9 of the 10 patients. Additionally, patients who had infections prior to receiving gene therapy have all recovered.

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