Development of cGMP packaging cell lines for retro & lentivirus production using innovative molecular engineering strategies

近年来，一系列自身免疫性疾病和癌症的治疗进展要求越来越复杂的医疗解决方案。一种迅速扩大的非常成功的治疗方法是将DNA输送到人类细胞（基因治疗），为它们提供新的特征和特性，以帮助对抗疾病。实现这一目标的一种高效方法是使用修饰过的病毒，如慢病毒，来传递DNA。然而，制造慢病毒的过程效率非常低，因为还没有制造出能够有效包装病毒的细胞。其原因是，完成这一过程所需的一些基因对细胞是有毒的。我们最近开发了一种新的解决方案来解决这个问题，并且已经产生了第一代能够高效生产慢病毒的细胞系。我们现在的目标是开发该细胞系的临床级版本，并创建一系列进一步，更先进的细胞系，以改善慢病毒的生产。

Recent advances in the treatment of a range of autoimmune diseases and cancer have requiredincreasingly complex medical solutions. One rapidly expanding range of very successful treatments isthe delivery of DNA to human cells (gene therapy) to provide them with new features and propertiesto help fight disease. A highly efficient method of achieving this is to use modified viruses, such aslentiviruses, to deliver the DNA. However, the process of making lentiviruses is highly inefficientbecause no cells have yet been made that allow the virus to be packaged efficiently. The reason forthis is that some of the genes required to do this are toxic to the cell. We have recently developed anovel solution to this problem, and have already generated a first generation cell line that produceslentiviruses highly efficiently. We now aim to develop clinical grade versions of this cell line andcreate a series of further, more advanced, cell lines for improved lentivirus production.