McTaggart Sally, Al-Rubeai Mohamed
Animal Cell Technology Group, School of Chemical Engineering, University of Birmingham, Edgbaston, Birmingham B15 2TT, UK.
Biotechnol Adv. 2002 Apr;20(1):1-31. doi: 10.1016/s0734-9750(01)00087-8.
The potential for gene therapy to cure a wide range of diseases has lead to high expectations and a great increase in research efforts in this area. At present, viral vectors are the most efficient means of delivering a corrective gene into human cells. While a number of different viral vectors are under development, retroviral vectors are currently the most common type used in clinical trials today. However, the production of retroviral vectors for gene therapy applications faces a number of challenges. Of primary concern is the low titre of vector stocks produced by packaging cells in culture and the inherent instability of retroviral vector activity. The problems facing large-scale retroviral vector production are outlined in this review and the research efforts by a number of groups who have attempted to optimise production methods are presented.
基因疗法治愈多种疾病的潜力引发了人们的高度期望,并促使该领域的研究工作大幅增加。目前,病毒载体是将校正基因导入人类细胞的最有效手段。虽然有多种不同的病毒载体正在研发中,但逆转录病毒载体是目前临床试验中最常用的类型。然而,用于基因治疗的逆转录病毒载体的生产面临诸多挑战。最主要的问题是培养中的包装细胞产生的载体储备滴度低以及逆转录病毒载体活性的固有不稳定性。本文综述了大规模生产逆转录病毒载体所面临的问题,并介绍了一些试图优化生产方法的研究团队所做的工作。