Molecular Immunology Unit,Institute of Child Health (ICH),University College London (UCL),30 Guilford Street,London WC1N 1EH,UK.
Expert Rev Mol Med. 2015 Nov 4;17:e19. doi: 10.1017/erm.2015.14.
Alongside advancements in gene therapy for inherited immune disorders, the need for effective alternative therapeutic options for other conditions has resulted in an expansion in the field of research for T cell gene therapy. T cells are easily obtained and can be induced to divide robustly ex vivo, a characteristic that allows them to be highly permissible to viral vector-mediated introduction of transgenes. Pioneering clinical trials targeting cancers and infectious diseases have provided safety and feasibility data and important information about persistence of engineered cells in vivo. Here, we review clinical experiences with γ-retroviral and lentiviral vectors and consider the potential of integrating transposon-based vectors as well as specific genome editing with designer nucleases in engineered T cell therapies.
除了遗传性免疫疾病的基因治疗进展之外，对于其他疾病的有效替代治疗选择的需求也促使 T 细胞基因治疗领域的研究不断扩展。T 细胞易于获取，并且可以在体外强烈诱导分裂，这一特性使其能够高度耐受病毒载体介导的转基因导入。针对癌症和传染病的开创性临床试验提供了安全性和可行性数据，以及关于工程化细胞在体内的持久性的重要信息。在这里，我们回顾了 γ-逆转录病毒和慢病毒载体的临床经验，并考虑了基于转座子的载体以及与设计性核酸酶的特定基因组编辑在工程 T 细胞治疗中的整合潜力。
