The efficacy of these cells, generated using lentiviral-mediated transduction of FOXP3, has been demonstrated in preclinical models of GvHD and IPEX, and is currently being evaluated in a clinical study of patients with IPEX syndrome (NCT05241444) (154, 157). The gene discussed is FOXP3; the disease is immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome.