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

基因疗法为你的细胞提供基因，以取代有缺陷的基因，或者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，通常情况下，成功的治疗需要许多不同的蛋白质(因此也就是基因)。这项工作旨在开发一种系统，通过该系统，多个基因可以串联在一起，形成一个单基因：多基因。通过基因疗法导入一个基因要比尝试将多个单独的基因导入一个细胞要容易得多，也更安全。这种新型的多基因能够产生所有不同的蛋白质(并且同时产生)的几率比许多单个基因的几率要高得多。我们希望在这里开发的方法将扩大可以用基因疗法治疗的疾病的范围。

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.