Optimisation of the 2A Co-expression System for Gene Therapies

基因疗法为你的细胞提供基因，要么取代有缺陷的基因，要么提供可以对抗其他形式疾病的基因。然后这些基因被翻译成蛋白质。然而，成功的治疗通常需要许多不同的蛋白质（因此也需要基因）。这项工作的目的是开发一个系统，使多个基因可以串在一起，形成一个单一的基因：多基因。通过基因治疗引入一个基因比试图将许多单个基因引入细胞更容易和安全。这种新型的多基因能够产生所有不同蛋白质的机会（并且所有蛋白质都在同一时间）比许多单个基因的情况要高得多。我们希望在这里开发的方法将扩大可以通过基因治疗治疗的疾病的范围。

Gene therapy supplies your cells with genes either to replace faulty ones, or to supply genes that can combat other forms of disease. These genes are then translated into proteins. Often, however, a number of different proteins (and hence genes) are required for successful therapy. This work is designed to develop a system whereby multiple genes can be strung together to form a single gene: a multi-gene. It is much easier and safer to introduce one gene via gene therapy, than try to introduce many individual genes into a cell. The chances of this new type of multi-gene being able to produce all of its different proteins (and all at the same time) is very much higher than is the case with many individual genes. The methods we wish to develop here will extend the range of diseases that may be treated by gene therapy.