Gene therapy approach restores immune function to children with rare immunodeficiency disorder

Researchers from the University of California Los Angeles (UCLA; CA, USA) and Great Ormond Street Hospital (GOSH; London, UK) have collaborated to develop a new gene therapy for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). The study, published in the New England Journal of Medicine, reports the 2- to 3-year outcomes of 50 children treated in clinical trials at GOSH, UCLA Mattel Children's Hospital and the National Institutes of Health between 2012 and 2017. The gene therapy approach used in the study involved the removal of blood-forming stem cells from the patients and the use...