Perry Wilson, MD, MSCE, Director of the Clinical and Translational Research Accelerator at Yale School of Medicine in New Haven, Connecticut, discusses gene therapy for children with severe combined immunodeficiency. SCID, or sometimes known as “bubble boy” syndrome, was a death sentence for children previously. A recent study using autologous ex vivo lentiviral gene therapy for adenosine deaminase deficiency resulted in 48 of 50 children achieving cure, with limited side effects, and notably, no development of cancer.