D'Costa Jenice, Mansfield S Gary, Humeau Laurent M
VIRxSYS Corp, Gaithersburg, MD 20877, USA.
Curr Opin Mol Ther. 2009 Oct;11(5):554-64.
Lentiviral vectors (LVs) are the most recently developed viral-derived vectors for gene therapy applications, and have demonstrated much promise. The ability to transduce dividing and non-dividing cells, and sustain long-term transgene expression makes LVs uniquely desirable as gene therapy vectors. With advances in vector design and large-scale production, LVs have become safer and more effective gene delivery systems. Since the first clinical trial was approved in 2002, several trials to treat patients with both infectious and genetic diseases have been approved. This review focuses on ongoing and planned trials of LV-based gene therapy.
慢病毒载体(LVs)是基因治疗应用中最新开发的病毒衍生载体,已展现出很大的前景。转导分裂细胞和非分裂细胞以及维持长期转基因表达的能力,使得慢病毒载体作为基因治疗载体极具吸引力。随着载体设计和大规模生产技术的进步,慢病毒载体已成为更安全、更有效的基因递送系统。自2002年首个临床试验获批以来,多项治疗感染性疾病和遗传性疾病患者的试验已获批准。本综述重点关注基于慢病毒载体的基因治疗正在进行的和计划中的试验。