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基因疗法:包治百病 – 译学馆
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基因疗法:包治百病

A new superweapon in the fight against cancer | Paula Hammond

癌症是我们每个人的心病
Cancer affects all of us —
尤其是那些不断复发
especially the ones that come back over and over again,
容易扩散 具有抗药性的癌症
the highly invasive and drug-resistant ones,
药物治疗对它们无效
the ones that defy medical treatment,
即使我们用最好的药也无济于事
even when we throw our best drugs at them.
在分子级别进行 基因工程改造
Engineering at the molecular level,
从最小结构层面着手
working at the smallest of scales,
(我们就)能找到意想不到的新办法
can provide exciting new ways
同最致命的癌症进行斗争
to fight the most aggressive forms of cancer.
癌症是一种很狡猾的疾病
Cancer is a very clever disease.
幸运的是 对于一些癌症
There are some forms of cancer,
我们可以通过一些 验证过的药物和手术
which, fortunately, we’ve learned how to address relatively well
进行治疗 可以得到相对较好的结果
with known and established drugs and surgery.
但有几种癌症
But there are some forms of cancer
这些方法对它们都无效
that don’t respond to these approaches,
即使在药物的猛烈攻击下
and the tumor survives or comes back,
肿瘤依然能够存活 或者复发
even after an onslaught of drugs.
我们可以将这几种致命的癌症
We can think of these very aggressive forms of cancer
想象成漫画书里面的大反派
as kind of supervillains in a comic book.
他们狡猾 适应性强
They’re clever, they’re adaptable,
生存能力一流
and they’re very good at staying alive.
而且跟现在漫画书里 大多数反派一样
And, like most supervillains these days,
他们的超能力来自基因突变
their superpowers come from a genetic mutation.
这些肿瘤细胞的基因不断变化
The genes that are modified inside these tumor cells
能找到新的、意想不到的生存方式
can enable and encode for new and unimagined modes of survival,
可以让癌细胞抵抗住
allowing the cancer cell to live through
我们最强大的化学治疗
even our best chemotherapy treatments.
有一个例子很神奇 基因能让细胞
One example is a trick in which a gene allows a cell,
在药物接近之前
even as the drug approaches the cell,
把药物推开
to push the drug out,
药物根本来不及起任何作用
before the drug can have any effect.
就好比细胞一口把药吐出去
Imagine — the cell effectively spits out the drug.
而这仅仅是我们的大反派癌症的
This is just one example of the many genetic tricks
众多基因把戏之一
in the bag of our supervillain, cancer.
这一切都跟突变基因有关
All due to mutant genes.
因此我们要面对的是拥有超强能力的大反派
So, we have a supervillain with incredible superpowers.
我们需要新的、更强大的攻击方式
And we need a new and powerful mode of attack.
实际上 我们可以关闭基因
Actually, we can turn off a gene.
开关是一系列叫siRNA的分子(即小分子干扰核糖核酸)
The key is a set of molecules known as siRNA.
它是一小段基因编码
siRNA are short sequences of genetic code
可以让细胞阻止某些特定基因(发挥作用)
that guide a cell to block a certain gene.
每一个siRNA都能关闭细胞内部
Each siRNA molecule can turn off a specific gene
某个特定的基因
inside the cell.
自siRNA被发现后
For many years since its discovery,
科学家们兴奋了好多年
scientists have been very excited
因为他们可以将这种基因拦截者加入药物中
about how we can apply these gene blockers in medicine.
但是有一个问题
But, there is a problem.
siRNA在细胞内部可以正常发挥作用
siRNA works well inside the cell.
但如果它接触到
But if it gets exposed to the enzymes
位于我们血液或者组织中的酶的话
that reside in our bloodstream or our tissues,
几秒钟就会被降解
it degrades within seconds.
因此它们必须被包裹保护起来 才能完成在人身体里的旅程
It has to be packaged, protected through its journey through the body
顺利抵达最终目标 癌细胞内部
on its way to the final target inside the cancer cell.
因此我们的策略如下
So, here’s our strategy.
首先 我们将siRNA基因拦截者 用在癌细胞身上
First, we’ll dose the cancer cell with siRNA, the gene blocker,
让(癌细胞的)生存基因失效
and silence those survival genes,
然后我们再用化学药品来对付癌细胞
and then we’ll whop it with a chemo drug.
但如何实现呢?
But how do we carry that out?
答案是分子工程技术
Using molecular engineering,
我们可以设计一种超级武器
we can actually design a superweapon
能在血液循环中存活下来
that can travel through the bloodstream.
它必须足够小 才可以进入血液循环
It has to be tiny enough to get through the bloodstream,
穿过肿瘤组织
it’s got to be small enough to penetrate the tumor tissue,
进入癌细胞内部
and it’s got to be tiny enough to be taken up inside the cancer cell.
要做到这一点
To do this job well,
它的尺寸必须小到头发的百分之一
it has to be about one one-hundredth the size of a human hair.
来仔细看一下我们是 如何制造这种纳米粒子的
Let’s take a closer look at how we can build this nanoparticle.
首先 我们要制造纳米核
First, let’s start with the nanoparticle core.
那是一颗小胶囊 内含化学药剂
It’s a tiny capsule that contains the chemotherapy drug.
对肿瘤细胞而言是致命毒药
This is the poison that will actually end the tumor cell’s life.
在内核周围我们包上一层很厚的
Around this core, we’ll wrap a very thin,
当然也就几纳米厚的siRNA
nanometers-thin blanket of siRNA.
这是我们的基因拦截者
This is our gene blocker.
因为siRNA带负电
Because siRNA is strongly negatively charged,
我们用一层
we can protect it
坚固的、带正电的聚合物来保护它
with a nice, protective layer of positively charged polymer.
这两种带有不同电荷的分子
The two oppositely charged molecules stick together
相互吸引
through charge attraction,
形成一层保护膜
and that provides us with a protective layer
防止siRNA在血液中被降解
that prevents the siRNA from degrading in the bloodstream.
别着急 我快说完了
We’re almost done.
(笑声)
(Laughter)
但前方还有一个大障碍需要跨越
But there is one more big obstacle we have to think about.
而且 也许是最大的一个障碍
In fact, it may be the biggest obstacle of all.
我们该如何部署这件超级武器?
How do we deploy this superweapon?
每一件武器都要设定目标
I mean, every good weapon needs to be targeted,
我们要将这件超级武器 瞄准潜伏在肿瘤内部的
we have to target this superweapon to the supervillain cells
超级大反派
that reside in the tumor.
但人体是有免疫系统的:
But our bodies have a natural immune-defense system:
血液里的各种细胞
cells that reside in the bloodstream
能辨认出外来物体
and pick out things that don’t belong,
然后杀死或清除它们
so that it can destroy or eliminate them.
很显然 我们的纳米粒子就是一种外来物体
And guess what? Our nanoparticle is considered a foreign object.
我们要让它骗过肿瘤的防御系统
We have to sneak our nanoparticle past the tumor defense system.
我们要伪装它 帮它通过重重岗哨
We have to get it past this mechanism of getting rid of the foreign object
进入敌营
by disguising it.
于是我们再加上一层 带负电的保护膜
So we add one more negatively charged layer
包住我们的纳米粒子
around this nanoparticle,
目的有两个
which serves two purposes.
第一 最外面这层保护膜是存在于我们体内的
First, this outer layer is one of the naturally charged,
带电多糖水合物之一
highly hydrated polysaccharides that resides in our body.
它能在纳米粒子周围形成许多水分子
It creates a cloud of water molecules around the nanoparticle
就像给纳米粒子穿上了隐形披风
that gives us an invisibility cloaking effect.
这件披风能帮助纳米粒子
This invisibility cloak allows the nanoparticle
在血液里自由行动
to travel through the bloodstream
畅通无阻
long and far enough to reach the tumor,
直到抵达肿瘤细胞
without getting eliminated by the body.
第二 这层保护膜含有一些分子
Second, this layer contains molecules
专门用来与肿瘤细胞结合
which bind specifically to our tumor cell.
一旦结合 癌细胞就会把纳米粒子吸收
Once bound, the cancer cell takes up the nanoparticle,
这样我们的纳米粒子最终就会进入癌细胞
and now we have our nanoparticle inside the cancer cell
准备发起进攻
and ready to deploy.
是啊!我也觉得很兴奋 继续!
Alright! I feel the same way. Let’s go!
(掌声)
(Applause)
siRNA首先开始行动
The siRNA is deployed first.
它需要几个小时
It acts for hours,
来让(癌细胞的)生存基因失效
giving enough time to silence and block those survival genes.
我们去除了(癌细胞的)基因超能力
We have now disabled those genetic superpowers.
只剩下没有防御的癌细胞
What remains is a cancer cell with no special defenses.
之后化学药剂从内核中释放出来
Then, the chemotherapy drug comes out of the core
干净利落地杀死肿瘤细胞
and destroys the tumor cell cleanly and efficiently.
只要有足够多的基因拦截者
With sufficient gene blockers,
我们就能对付各种变异的癌细胞
we can address many different kinds of mutations,
有机会消灭肿瘤
allowing the chance to sweep out tumors,
不留落网之鱼
without leaving behind any bad guys.
那么 我们的方法到底有没有效呢?
So, how does our strategy work?
我们进行了动物实验
We’ve tested these nanostructure particles in animals
使用的是一种非常厉害的癌症 三阴性乳腺癌
using a highly aggressive form of triple-negative breast cancer.
它的基因非常厉害
This triple-negative breast cancer exhibits the gene
能迅速将抗癌药排出
that spits out cancer drug as soon as it is delivered.
通常 我们会使用阿霉素这种抗癌药
Usually, doxorubicin — let’s call it “dox” — is the cancer drug
它是治疗乳腺癌的首选手段
that is the first line of treatment for breast cancer.
第一次 我们只用阿霉素来对动物进行治疗
So, we first treated our animals with a dox core, dox only.
肿瘤的增长率有所减缓
The tumor slowed their rate of growth,
但还是增长很快
but they still grew rapidly,
两周之内就增长了一倍
doubling in size over a period of two weeks.
接下来 我们的超级武器上场了
Then, we tried our combination superweapon.
外层是siRNA 保护自身不被拦截
A nanolayer particle with siRNA against the chemo pump,
内核部分是阿霉素
plus, we have the dox in the core.
结果我们发现 肿瘤不但没有增长
And look — we found that not only did the tumors stop growing,
而且变小了
they actually decreased in size
在某些样本上甚至完全消失
and were eliminated in some cases.
肿瘤被击退了
The tumors were actually regressing.
(掌声)
(Applause)
这种方法最大的好处就是可以定制
What’s great about this approach is that it can be personalized.
我们可以添加多重siRNA
We can add many different layers of siRNA
分别针对不同的变异和防御机制
to address different mutations and tumor defense mechanisms.
我们还可以在内核中 放入不同种类的药物
And we can put different drugs into the nanoparticle core.
随着医生掌握测试方法
As doctors learn how to test patients
弄清肿瘤的特定基因类型
and understand certain tumor genetic types,
就能帮我们确定 哪些病人能使用这种疗法
they can help us determine which patients can benefit from this strategy
应该选用哪种基因拦截者
and which gene blockers we can use.
我想特别说说卵巢癌
Ovarian cancer strikes a special chord with me.
它是一种非常可怕的癌症
It is a very aggressive cancer,
一部分原因是它只有在晚期才能被发现
in part because it’s discovered at very late stages,
那时病情已经很严重
when it’s highly advanced
而且伴随很多基因突变
and there are a number of genetic mutations.
在首轮化疗结束之后
After the first round of chemotherapy,
有75%的患者会复发
this cancer comes back for 75 percent of patients.
而且复发之后通常都有抗药性
And it usually comes back in a drug-resistant form.
晚期卵巢癌
High-grade ovarian cancer
是最大的超级反派之一
is one of the biggest supervillains out there.
它是我们的超级武器
And we’re now directing our superweapon
下一个目标
toward its defeat.
作为一名研究者
As a researcher,
我通常很少与病人接触
I usually don’t get to work with patients.
但最近我遇到了一位母亲
But I recently met a mother
她叫咪咪是一名卵巢癌幸存者 她有个女儿叫佩吉
who is an ovarian cancer survivor, Mimi, and her daughter, Paige.
她们母女俩表现出来的乐观和坚强
I was deeply inspired by the optimism and strength
勇气和相互支持
that both mother and daughter displayed
深深地打动了我
and by their story of courage and support.
我们聊到了用于治疗癌症的
At this event, we spoke about the different technologies
科技手段
directed at cancer.
咪咪听得流泪了
And Mimi was in tears
她说了解到目前取得的这些成就
as she explained how learning about these efforts
让她看到了下一代人(不受癌症折磨)的希望
gives her hope for future generations,
其中包括她的女儿
including her own daughter.
这让我很是感慨
This really touched me.
(我们所做的)不仅仅是建立一门优秀的科学
It’s not just about building really elegant science.
而是在改变人的命运
It’s about changing people’s lives.
是要了解分子工程学
It’s about understanding the power of engineering
强大的力量
on the scale of molecules.
我坚信像佩吉这样的学生 只要在这条路走下去
I know that as students like Paige move forward in their careers,
他们一定能打开一片新天地
they’ll open new possibilities
解决当今世界最大的健康问题
in addressing some of the big health problems in the world —
包括卵巢癌、神经系统疾病、传染病
including ovarian cancer, neurological disorders, infectious disease —
就如同化学基因改造 已经为我指明了一条道路
just as chemical engineering has found a way to open doors for me,
提供了一种基因改造的方法
and has provided a way of engineering
让我们可以从小小的分子入手
on the tiniest scale, that of molecules,
解决人类健康这个大问题
to heal on the human scale.
谢谢大家
Thank you.
(掌声)
(Applause)

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视频概述

疑难杂症对我们来说是什么?可以这样形容:它是一种非常狡猾的死神,悄无声息地偷走我们的健康,如何打败死神呢,不用怕,我有基因超能力?!

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赖皮

视频来源

https://www.youtube.com/watch?v=1N39Z0ODeME

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