Lehn P
Laboratoire de Biochimie Génétique, Université René Descartes, CHU Paris-Ouest, Hôpital Robert Debré, France.
Pathol Biol (Paris). 1993 Oct;41(8):658-62.
This paper describes the various strategies for somatic gene therapy. There are two main scenarios: the ex vivo approach and the in vivo approach. As recombinant retroviruses were used in the first clinical trials of gene therapy, their main characteristics (structure, efficient production of helper-free viral stocks by using packaging cells, transduction procedures) are discussed as well as the related safety issues. Finally, retroviral vectors can also be used as a new tool to investigate various clinical situations as they allow to mark cells by stably integrating in the cellular genome.
本文描述了体细胞基因治疗的各种策略。主要有两种情况：体外方法和体内方法。由于重组逆转录病毒被用于基因治疗的首次临床试验，因此讨论了它们的主要特征（结构、通过使用包装细胞高效生产无辅助病毒原液、转导程序）以及相关的安全问题。最后，逆转录病毒载体也可以用作研究各种临床情况的新工具，因为它们能够通过稳定整合到细胞基因组中来标记细胞。
