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利用逆转录病毒载体系向体细胞中转移外源基因，具有高效、低拷贝整合和安全的特点。是进行基因治疗的理想的工具。1990年美国NIH的Blases等人获准利用这一技术将腺苷脱氨酶（ADA）基因导入一因ADA缺陷所致的重症联合免疫缺损病孩的淋巴细胞中，将这些经过基因改造的淋巴细胞回输给患者，其ADA水平大幅度上升，免疫系统的功能得到了有效的恢复，“治疗效果出乎意外的好。”这将是人类历史上第一例成功的基因治疗的病例。
The use of retroviral delivery systems to transfer foreign genes to somatic cells has the characteristics of high efficiency, low copy integration, and safety. It is an ideal tool for gene therapy. In 1990, Blases et al. of NIH in the United States were allowed to use this technology to introduce the adenosine deaminase (ADA) gene into lymphocytes of a severe combined immunodeficiency disease child due to ADA deficiency, and to use these genetically modified lymphocytes. In return to patients, their ADA levels have risen dramatically and the immune system has been effectively restored. “The treatment effect is unexpectedly good.” This will be the first case of successful gene therapy in human history.