Gene Therapy Restores Sight In Mice

fungyun

http://english.ntdtv.com/ntdtv_e ... 7/518044545351.html

This mouse is blind. Slowed down by water for proper observation, the mouse can't find its way towards light.

It suffers from Retinitus Pigmentosa, a genetic disease that affects the eye's ability to communicate with the brain. The disease affects an estimated two million people in the United States alone.

Alan Horsager, a professor of neuroscience at USC, is using an experimental gene therapy that delivers light-activated proteins to the cells of the mouses' retinas. He's hoping the proteins will create new photoreceptor
cells which will allow the mouse to see.

He doesn't have to wait long before the mouse begins to respond exactly as intended.

[Alan Horsager,  Neuroscience Professor, University of Southern California]:

"What we find is that these mice that are originally blind, these are models
that represent retinitis pigmentosa in people, actually have restored visual
function."

The success of the experiments has spurred Horsager to form EOS
Neuroscience - a company that aims to commercialize treatment for people who
suffer from retinal disease. The treatment is based on optogenetics - an
emerging field of research that uses light to control genetically engineered
cells.

Ed Boyden, an assistant professor at MIT, is one of the pioneers of
optogenetics. His specialty is the study and potential control of neurons,
the cells that comprise the brain.

[Ed Boyden, Assistant Professor, Massachusetts Institue of Technology]:

"What we like to be able to do and what we have started to be able to do is
to use light to turn on and off each of these regions and then pathways and
cell types within those pathways in order to figure out how they work
together. So we turn something off we can figure out what it was needed for,
we have deleted it and we can assess its necessity. And by stimulating
something we can figure out what its power could be, what it could do
causally to its neighbors."

In his lab he uses rats to show that by engineering neurons to become
sensitive to light, he can target and control different parts of their
brains.

Ultimately, he says, his research will be applicable to humans.

The human brain is made up of billions of neurons, all inter-connected to
form a complex network that scientists are just now starting to understand.

[Ed Boyden, Assistant Professor, Massachusetts Institute of Technology]:

"If we think about the complexity of the brain, only a very small portion
has been studied in very great detail. What we would like to do is really
reveal the principles of how to control these circuits to fix their problems
when they go awry."

Boyden says optogenetics could eventually lead to treatments or even cures,
for neurological disorders like Parkinson's and Alzheimer's.

While the research is still in its infancy, Alan Horsager believes that
optogenetics has the potential to restore vision in people, as it has in his
lab mice, in the near future. He hopes to start clinical trial on humans in
a couple of years.

Both he and Ed Boyden believe that curing some forms of blindness will be
the first of many benefits to come from this emerging field.

雪山飞狐

谢谢楼主的分享！

flyeagle

看样子是个好消息，可是，我小学没毕业，看不懂，有哪位高手翻译一下，

衰神附体

有道翻译的：
这只老鼠是盲目的。以水为适当放慢脚步观察,鼠标找不到它的方式走向光明。


它有Retinitus Pigmentosa,一种遗传性疾病,影响眼球的沟通能力与大脑。该疾病影响估计有200万人仅在美国。


艾伦Horsager教授在USC的神经系统,使用一个实验性基因治疗,提供一种可由光来激活蛋白和细胞的老鼠的视网膜。他希望将创造新的感光细胞的蛋白质

这将使得老鼠细胞要看的东西。


他没有时间去等待鼠标开始回应前完全一样的本意。


阿伦Horsager、神经系统科学教授,南加州大学):


“我们发现的是,这些小鼠,原来是盲目的,这些都是模型

在人们表示患有色素性视网膜炎,实际上是有恢复视觉

功能。”


实验的成功,促使Horsager形成EOS

神经科学——一个公司,旨在商业化治疗的人

患有视网膜疾病。基于optogenetics治疗——一个

新兴的研究领域,利用光来控制基因工程

细胞。


艾德Boyden,助理教授在麻省理工学院,是时代的先驱

optogenetics。他的专长是研究和潜在的控制神经元,

大脑细胞所组成的。


(比如Boyden、助理教授、麻萨诸塞州科技公司]:


“我们希望能够如此做的,什么是我们已经开始能够做的就是

使用光开通和关每一个这些区域中,然后途径和

细胞类型,只要在这些通路为了找出他们是如何工作的

在一起。所以我们把东西掉我们可以找出哪些是需要的,

我们已经删除了它,我们可以评估其必要性。和通过刺激

我们可以想出什么东西,它的力量能被它所能做的一切

向其邻国而存在。”


在他的实验室里,他使用大鼠神经元来表明由工程成为什么样的人

敏感的光,他可以针对不同的部分和控制

大脑。


最终,他说,他的研究将适用于人类。


人类的大脑是由数十亿个神经元,所有互相连接

组成一个复杂的网络系统,科学家正开始理解了。


(比如Boyden、助理教授、麻省理工学院]:


“如果我们想错综复杂的大脑,只有一小部分

已经研究了非常详细。我们想要做的事是真的

揭示了如何控制这些原理电路固定他们的问题

当他们的出错率有多高。”


Boyden说optogenetics,最终可能导致治疗甚至疗法,

神经学疾病如帕金森氏症和阿兹海默症。


同时研究还处于初级阶段,阿兰Horsager相信

optogenetics有潜力恢复视力在人们的,因为它已经在他的

实验室的老鼠,在不久的将来。他希望在人类开始在临床试验

上好几年的时间。


艾德Boyden,他也相信固化某些形式的失明

第一次的许多好处来自于这个新兴领域。

flyeagle

4# 衰神附体
哈哈，高手，不过里面的鼠标应该全改成实验鼠。谢谢您为大家做出的贡献。

衰神附体

哈哈，有道翻译笨的了