Well, I talked about the problem of using plasmids in gene therapy, that it's very difficult to get them into cells.
我们之前讨论过,基因治疗中运用质粒的问题,质粒本身极难进入细胞
If you can genetically engineer viruses like this and viruses have all those good properties as gene therapy vehicles that I described.
如果你能像我说的这样在遗传上改造病毒,加之病毒具备诸多优点,适合作为基因治疗的载体
There's a variety of different ways to think about using gene therapy and they're illustrated in this picture.
有很多种,基因治疗的思路,这张图上有图示
So, making gene therapy specific is a key problem in making it work.
所以基因治疗中,特异性对能否成功就至关重要了
Those are some example of different strategies for using gene therapy in people.
以上就是几种,人类基因治疗的不同策略
Because of this, because cystic fibrosis is a disease that's caused by a single gene defect, there's been great interest in trying to treat cystic fibrosis with gene therapy.
就是因为如此,就因为囊胞性纤维症,是一种单基因病,人们对于用基因治疗,治愈囊胞性纤维症产生了很大的兴趣
That's another advantage of using viruses as a gene therapy vector.
这是病毒,作为基因疗法载体的另一个优势
Why isn't that the solution to gene therapy?
但病毒为什么不是基因治疗的解决方案呢
This is a common problem in gene therapy.
这是基因治疗中的通病
That is also an advantage of using them as gene therapy vehicles because they will only infect cells that they are - that they have an affinity for or that they're prone to affect.
非常有利于将其,作为基因治疗的载体使用,因为它们只能感染那些--,它们对之具有亲和性,或者说它们倾向于去感染的细胞
Before we talk about how to do it-- and we don't have perfect ways to do it yet but I'll describe some of the ways that are used, I want to talk just briefly on this slide about what the goals for gene therapy might be.
在讲具体怎么做之前--,现在还没有任何一种方法是完美的,但我还是要讲讲一些已经应用的方法,我借助这张幻灯片简要介绍一下,基因治疗的可能目标
I talked about just not using viruses but using plasmid DNA together with lipids and that's another strategy for gene therapy.
这一部分我们不仅讲了病毒的应用,也讲了利用脂质体包裹的质粒DNA,基因治疗还有其它的策略
We'll start with Week 2 talking about Genetic Engineering; what's DNA, how can it be manipulated, how is our ability to manipulate DNA led to things like gene therapy which can now be done in people.
从第二周开始我们会讲基因工程,什么是DNA,怎么样才能操控它们,我们如何操作DNA,并将它应用于人类的基因疗法
So in the first week we run a section called from strawberries to gene therapy where we talk about DNA, extract DNA, you can play with the DNA of an organism and we can think about how to use DNA for other purposes.
第一周组会的主题是从草莓到基因疗法,主要讨论DNA和DNA的提取,使你们对有机组织的DNA有直观的认识,并思考DNA的其它应用
But if you're trying to use that virus as a gene delivery vector and your body gets rid of it then the therapy has failed, it's ended.
但如果你想把腺病毒作为基因治疗载体,而你的身体清除这种病毒,治疗就失败了,前功尽弃了
Then you could imagine blocking gene expression would be a therapy.
这样你们就会认为,阻止基因的表达是一种治疗手段了
应用推荐