Lentivirus technologies for modulation of the immune system
Lentivirus technologies for modulation of the immune system
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DOI：
10.1016/j.coph.2015.08.007
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发表时间：
2015-10-01
影响因子：
4
通讯作者：
Thrasher, Adrian J.
中科院分区：
文献类型：
--
作者：
Houghton, Benjamin C.;Booth, Claire;Thrasher, Adrian J.
慢病毒载体（LVV）是治疗免疫系统疾病的重要工具。使用LW将治疗性遗传物质整合到造血干细胞区室中可以介导造血谱系的长期校正，从而校正疾病表型。二十年的载体开发已经成功地将LW带到了临床，现在正在报道治疗原发性免疫缺陷的临床试验的后续研究。结果表明，在没有早期逆转录病毒基因治疗试验中观察到的严重不良事件的情况下，患有多种疾病的患者的生活质量得到了明显改善。对基因修饰的过继性T细胞转移作为替代策略的兴趣日益增长，推动了进一步的技术创新，包括新型病毒包膜的表征。我们还将讨论基因编辑技术在免疫缺陷模型中的临床前研究进展。
Lentiviral vectors (LVV) are important tools for the treatment of immune system disorders. Integration of therapeutic genetic material into the haematopoietic stem cell compartment using LW can mediate long-term correction of haematopoietic lineages, thereby correcting disease phenotypes. Twenty years of vector development have successfully brought LW to the clinic, with follow up studies of clinical trials treating primary immunodeficiencies now being reported. Results have demonstrated clear improvements in the quality of life for patients with a number of conditions in the absence of the severe adverse events observed in earlier retroviral gene therapy trials. Growing interest in gene modified adoptive T cell transfer as an alternative strategy has driven further technology innovation, including characterisation of novel viral envelopes. We will also discuss the progression of gene editing technology to preclinical investigations in models of immune deficiency.