Investigating pseudotyped lentiviral vector for gene delivery to the lung.

基因疗法有可能治疗许多遗传或获得性疾病的根本原因。慢病毒载体提供了一种将基因（转基因）引入细胞的方法，使其整合到宿主细胞基因组中。这种整合提供了长期表达的可能性，因为慢病毒能够转导分裂细胞和非分裂细胞。在这个项目中，目的是评估两组不同的伪病毒（来自仙台病毒的F/HN和来自流感病毒的HA/NA）转导肺细胞的能力。该项目将比较这些分子组合的能力，当它们在相同的慢病毒载体上进行假型时，在肺内转导不同类型的细胞，并比较它们转导长时间表达转基因的细胞的能力。根据初步结果，可以修改载体成分，以便根据成功转导的细胞类型决定的特定肺部疾病优化基因转移到肺部。然后可以生成治疗性表达载体，以便在扩大规模和临床试验之前进行评估。

Gene therapy has the potential to treat the underlying cause of many inherited or acquired diseases. Lentiviral vectors offer a way of introducing a gene (transgene) into cells such that it is integrated into the host cell genome. This integration provides the possibility of long term expression as lentiviruses are capable of transducing both dividing and non-dividing cells. In this project the aim is to assess ability of two different sets of pseudotypes to transduce the cells of the lung (F/HN from the Sendai virus and HA/NA from the Influenza virus). The project will compare the ability of these sets of molecules, when pseudotyped onto the same lentiviral vector, to transduce various cell types within the lung and compare their ability to transduce cells that express the transgene for a long period of time. Depending on initial results the vector components may be modified in order to optimise gene transfer to the lungs for a specific lung disease decided upon based on cell types being successfully transduced. A therapeutic expressing vector may then be generated to carry forward for evaluation prior to scale up and clinical trials.