A gene therapy appears to be effective in treating severe combined immunodeficiency syndrome, or SCID, the Associated Press reports.
Researchers from the University of California, Los Angeles, and Great Ormond Street Hospital treated 50 children with SCID due to adenosine deaminase deficiency. ADA-SCID is caused by mutations in the ADA gene, which leads to an immune system that does not function properly and can often be fatal. As they report in the New England Journal of Medicine, the researchers used lentiviral vector-based gene therapy to deliver a working version of the gene to the children in their study.
Overall, 48 of the 50 children had restored ADA enzyme and immune system function following treatment and through 24 to 36 months of follow up. "They're no longer patients. They're off all of their antibiotics. They're living normal lives," senior author Donald Kohn from UCLA tells HealthDay.
The AP notes that it not yet clear how long the effect will last, but Stephen Gottschalk of St. Jude Children's Research Hospital, who was not involved in the study, tells it that the "immune function seems stable over time so I think it looks very, very encouraging."