Although no adverse events were seen, we wanted to evaluate whether transfer of gene-modified T cells and specifically CD8 T cells could correct the disease phenotype, especially because functional CD8 T cells are critical determinants of disease protection.14, 15, 17 The use of autologous gene-modified T cells also has an established safety profile, with hundreds of patients treated to date for hematologic malignancies in cancer immunotherapy trials with no reported transformational events. Here, CD8A is linked to hematologic disorder.