Improving Gene Transfer Into Haemopoietic Cells

改善造血细胞的基因转移

• 批准号: nhmrc : 115100
• 负责人: Prof John Rasko
• 金额: $ 13.03万
• 依托单位国家: 澳大利亚
• 项目类别: NHMRC Project Grants
• 财政年份: 2000
• 资助国家: 澳大利亚
• 起止时间: 2000-01-01 至 2002-12-31
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/improving-gene-transfer-haemopoietic-cells/101236
• 关键词: Improving; Gene; Transfer; Into; Haemopoietic

Gene therapy is a novel form of medical treatment in which healthy genes are used to replace defective genes in cells. It is sobering to realise that in the next few years the whole human DNA sequence will be known. Consequently the already large list of genes which are known to cause disease will be greatly expanded through the application of molecular genetics. Surprisingly, however, treatments based on the correction of disease genes in the cells of a patient are not keeping up with expectations. In attempting to achieve clinically relevant results, viruses (masters of forcing infected cells to do their bidding) have been harnessed to deliver healthy genes into diseased cells. The problem has been that the modified, safe viruses used clinically have not been efficient at achieving sustained production of healthy genes in sufficient numbers of cells. In the studies described , we will attack this problem using a number of different, but complementary approaches. Our main focus will be to facilitate efficient virus entry of appropriate target cells. We have recently been successful in cloning the receptors for two important viruses which can enter human cells. Identification of these receptors gives us clues to methods of improving virus entry. Now that we know the identity of these receptors, we can create tools to define the type of cells that these viruses can readily target.

基因治疗是一种新的医学治疗形式，其中健康的基因被用来取代细胞中的缺陷基因。我们清醒地认识到，在未来几年内，整个人类DNA序列将被发现。因此，通过分子遗传学的应用，已知引起疾病的基因的数量将大大增加。然而，令人惊讶的是，基于纠正患者细胞中疾病基因的治疗方法并没有达到预期。在试图获得临床相关结果的过程中，病毒（迫使受感染细胞服从命令的大师）被用来将健康基因传递到患病细胞中。问题是，临床上使用的改良的安全病毒在足够数量的细胞中持续产生健康基因方面并不有效。在所描述的研究中，我们将使用许多不同但互补的方法来解决这个问题。我们的主要重点将是促进有效的病毒进入适当的靶细胞。我们最近成功地克隆了两种能进入人体细胞的重要病毒的受体。这些受体的鉴定为我们提供了改善病毒进入的方法的线索。现在我们知道了这些受体的身份，我们可以创建工具来定义这些病毒可以容易靶向的细胞类型。