逆转录病毒
医学
严重联合免疫缺陷
载体（分子生物学）
病毒载体
病毒学
免疫缺陷
遗传增强
白血病
病毒
小鼠白血病病毒
免疫学
生物
体内
重组DNA
免疫系统
基因
生物技术
生物化学
作者
Salima Hacein‐Bey‐Abina,Sung‐Yun Pai,H. Bobby Gaspar,Myriam Armant,Charles C. Berry,Stéphane Blanche,Jack Bleesing,Johanna Blondeau,Helen de Boer,Karen Buckland,Laure Caccavelli,Guilhem Cros,Satiro De Oliveira,Karen S. Fernández,Dongjing Guo,Chad E. Harris,Gregory Hopkins,Leslie E. Lehmann,Apiradee Lim,Wendy B. London
标识
DOI：10.1056/nejmoa1404588
摘要
In previous clinical trials involving children with X-linked severe combined immunodeficiency (SCID-X1), a Moloney murine leukemia virus-based γ-retrovirus vector expressing interleukin-2 receptor γ-chain (γc) complementary DNA successfully restored immunity in most patients but resulted in vector-induced leukemia through enhancer-mediated mutagenesis in 25% of patients. We assessed the efficacy and safety of a self-inactivating retrovirus for the treatment of SCID-X1.
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(2025-6-4)
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