Using a defective retroviral vector, the investigators transduced ex vivo CD34+ bone marrow cells from five boys with this SCID, resulting in no adverse effects.

• 调查者使用一种有缺陷的逆转录病毒载体，把5名重症联合免疫缺陷患儿的CD34+骨髓细胞取出体外并进行转换。