Ghosh Sujal, Gaspar H Bobby
Infection, Immunity, Inflammation, Molecular and Cellular Immunology Section, University College London, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK; Department of Pediatric Oncology, Hematology and Clinical Immunology, Center of Child and Adolescent Health, Heinrich-Heine-University, Moorenstraße 5, 40225 Düsseldorf, Germany.
Infection, Immunity, Inflammation, Molecular and Cellular Immunology Section, University College London, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK.
Hematol Oncol Clin North Am. 2017 Oct;31(5):823-834. doi: 10.1016/j.hoc.2017.05.003. Epub 2017 Jun 29.
Transfer of gene-corrected autologous hematopoietic stem cells in patients with primary immunodeficiencies has emerged as a new therapeutic approach. Patients with various conditions lacking a suitable donor have been treated with retroviral vectors and a gene-addition strategy. Initial promising results were shadowed by the occurrence of malignancies in some of these patients. Current trials, developed in the last decade, use safer viral vectors to overcome the risk of genotoxicity and have led to improved clinical outcomes. This review reflects the progresses made in specific disorders, including adenosine deaminase deficiency, X-linked severe combined immunodeficiency, chronic granulomatous disease, and Wiskott-Aldrich syndrome.
基因校正自体造血干细胞移植已成为原发性免疫缺陷患者的一种新治疗方法。各种缺乏合适供体的患者已接受逆转录病毒载体和基因添加策略的治疗。这些患者中一些人发生恶性肿瘤的情况使最初的喜人结果蒙上了阴影。过去十年开展的当前试验使用更安全的病毒载体来克服基因毒性风险，并带来了更好的临床结果。本综述反映了在特定疾病中取得的进展，包括腺苷脱氨酶缺乏症、X连锁重症联合免疫缺陷、慢性肉芽肿病和威斯科特-奥尔德里奇综合征。
