TEHRAN, Young Journalists Club (YJC) - The revolutionary new gene therapy was developed in tandem between St. Jude Children's Research Hospital in Memphis, Tennessee, and the University of California San Francisco (UCSF) Benioff Children's Hospital, and the results were published in the New England Journal of Medicine on Thursday.
Based on a study of eight children that began when they were infants, the doctors found that by editing a portion of the patients' blood stem cells and adding a genetically modified HIV virus, seven of them were able to develop normal immune systems that could fight infections and even receive vaccines, something they hadn't been able to do previously. The procedure took between six and 24 months to be fully effective.
The children were all born with severe combined immunodeficiency syndrome (SCID), a genetic defect in which the bone marrow, which is the body's blood cell factory, fails to produce functional immune system cells able to combat infections. It affects 1 in 200,000 newborns, almost exclusively males.
Most infants born with SCID will die within a year or two of their birth.
This therapy has cured the patients," Ewelina Mamcarz, of the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy, said in a Wednesday press release. She noted that more observation is necessary, though.
"These patients are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives." Mamcarz noted. "This is a first for patients with SCID-X1."