The widespread clinical implementation of gene therapy requires the ability to stably integrate genetic information through gene transfer vectors in a safe, effective, and economical manner. The latest generation of Sleeping Beauty (SB) transposon vectors fulfills these requirements, and may overcome limitations associated with viral gene transfer vectors and transient nonviral gene delivery approaches that are prevalent in ongoing clinical trials. The SB system enables high-level stable gene transfer and sustained transgene expression in multiple primary human somatic cell types, thereby representing a highly attractive gene transfer strategy for clinical use. Here, we review the most important aspects of using SB for gene therapy, including vectorization as well as genomic integration features. We also illustrate the path to successful clinical implementation by highlighting the application of chimeric antigen receptor (CAR)-modified T cells in cancer immunotherapy.

基因治疗的广泛临床实施要求能够以安全，有效和经济的方式通过基因转移载体稳定整合遗传信息的能力。最新一代的睡美人（SB）转座子载体可以满足这些要求，并且可以克服与病毒基因转移载体和瞬态非病毒基因递送方法（正在进行的临床试验中普遍存在）相关的局限性。SB系统可在多种原代人体细胞类型中实现高水平的稳定基因转移和持续的转基因表达，从而代表了一种极具吸引力的临床基因转移策略。在这里，我们回顾了使用SB进行基因治疗的最重要方面，包括向量化以及基因组整合功能。我们还通过强调嵌合抗原受体（CAR）修饰的T细胞在癌症免疫治疗中的应用来说明成功实现临床的途径。