Department of Pediatrics, Hokkaido University Graduate School of Medicine, N15, W7, Kita-ku Sapporo, 060-8638, Japan.
Expert Rev Clin Immunol. 2013 Nov;9(11):1015-8. doi: 10.1586/1744666X.2013.850416.
Wiskott-Aldrich syndrome (WAS), an X-linked primary immunodeficiency disease (PID) with unique and characteristic features, had been considered to be a good candidate for gene therapy. In 2010, hematopoietic stem cell (HSC) gene therapy, using a retroviral vector, was performed for WAS patients; however, concerns remain regarding the long-term safety of this therapy as several patients with PID developed myeloproliferative diseases due to insertional mutagenesis related to HSC gene therapy using retroviral vectors. Aiuti et al. first reported HSC gene therapy for WAS using a lentiviral vector and compared the safety and efficacy of the two therapies in the context of the same disease background. They undertook a detailed study of the vector integration sites and concluded that lentiviral HSC gene therapy was safer than retroviral gene therapy.
威斯科特-奥尔德里奇综合征（Wiskott-Aldrich syndrome，WAS）是一种 X 连锁的原发性免疫缺陷病（PID），具有独特和特征性的特征，被认为是基因治疗的良好候选者。2010 年，采用逆转录病毒载体对 WAS 患者进行了造血干细胞（HSC）基因治疗；然而，由于使用逆转录病毒载体的 HSC 基因治疗引起与插入突变相关的骨髓增生性疾病，人们对这种治疗的长期安全性仍存在担忧。Aiuti 等人首次报道了使用慢病毒载体对 WAS 进行的 HSC 基因治疗，并在相同疾病背景下比较了两种治疗方法的安全性和疗效。他们对载体整合位点进行了详细研究，得出结论认为慢病毒 HSC 基因治疗比逆转录病毒基因治疗更安全。
