Elimination of packaging sequences from retroviruses to produce improved vectors for gene delivery

将基因插入细胞的能力可用于研究基因的作用，产生有用的蛋白质，如抗体，以及纠正遗传疾病患者的突变细胞。病毒可以作为基因传递载体（或“载体”），因为它们已经进化出了将基因插入细胞的能力。我们使用一种改良的HIV病毒，这种病毒不能繁殖或引起疾病，但可以将基因传递给细胞。这种载体在世界各地的实验室中使用，但有一些缺点，例如携带约18%的艾滋病毒基因组，并且可以传递的基因大小有限。我们已经开发出一种名为LTR1的流线型艾滋病毒载体，它只携带3%的艾滋病毒基因组。我们认为LTR1在安全性和效率方面可能是先前HIV载体的改进，因此希望将它们同时进行测试。我们将获得LTR1的商业许可，以便尽可能多的研究人员和工业合作伙伴可以使用这项技术突破。

The ability to insert genes into cells can be used to study the roles of genes, produce useful proteins like antibodies and correct mutated cells in patients with genetic disorders. Viruses can act as gene delivery vehicles (or 'vectors') because they have evolved the ability to insert genes into cells. We use a modified HIV virus which is unable to reproduce or cause disease but can deliver genes to cells. This vector is used in laboratories across the world but has some drawbacks such as carrying about 18% of the HIV genome and being limited in terms of the size of gene it can deliver. We have already developed a streamlined HIV vector called LTR1 which carries only 3% of the HIV genome. We believe that LTR1 may be an improvement on the previous HIV vector in terms of safety and efficiency so would like to test them side by side. We will licence LTR1 commercially so that this technological breakthrough will be available to as many researchers and industrial partners as possible.