
Chinese: 
近几十年来在基因治疗领域的研究正在开始取得回报。
2017年，第一个基因疗法产品在美国获得批准，
今年预计会有更多产品得到批准，并且同时还有数以千计的产品在准备临床试验，
其中就包括一项针对雷特综合征的临床试验，
由诺华(Novartis)旗下的AveXis公司资助进行，预计将于今年(2019年)开展。
那么，到底什么是基因疗法？
基因疗法是将一个新的基因加入到人体细胞中
替换掉错误的基因，以用来防止、改善或治愈疾病。
基因需要一些帮助才能进入到细胞中。
其中一种方法是通过病毒。
现在，想想病毒通常是怎么做的。
他们非常善于进入到我们的身体内，并让我们生病。
他们做到这一切的方法是将自己的基因物质注入到我们体内。

English: 
Decades of research in the gene therapy
field is paying off in 2017. The very
first gene therapy products were
approved in the US, with more likely to
be approved this year and thousands more
in clinical trials including a trial for
Rett syndrome sponsored by AveXis, a
Novartis company, expected to get
underway this year so what exactly is
gene therapy it's the addition of new
genes to a person cells to replace
faulty genes in an attempt to prevent
improve or cure disease genes need a bit
of help to get into cells one way they
get that help is through a virus now
think about what virus is normally do
they are really good at getting into our
bodies and making us sick they do that
by injecting their genetic material into
ourselves so scientists had the great

Chinese: 
因此科学家们就有了一个绝妙的主意，即通过劫持病毒，让它们把基因输送到细胞内。
不过不要担心，科学家们会移除掉那些能导致我们生病的部分。
这种修改后的病毒称为载体(vector)。
那么，对于雷特综合征来说这些是怎么工作的？
我们知道雷特综合征是由一个名为MECP2的基因发生突变导致的，
这种基因能够产生一种对大脑功能来说非常重要的蛋白质。
所以首先，上亿个MECP2基因的拷贝会在实验室中被生产出来，
然后这些生产出来的基因被装入到载体中。
在将要开展的临床试验中，
雷特患者将接受一种治疗，一次性将名为AVXS-201的基因疗法产品注射进脑脊髓液。
载体然后会将他们搭载的珍贵物质带入大脑中，
然后将这些物质输送到细胞的细胞核中。
一旦输送完成，载体会自然分解，
然后新的MECP2基因将会开始工作，制造健康的且重要的蛋白质。
我们的期望是接受治疗的个体能够显著改善他们的状况。

English: 
idea of hijacking a virus to deliver
genes into cells but don't worry they
remove the parts that make us sick the
modified virus is called a vector so how
would this work in Rett syndrome well we
know that ret is caused by mutations in
a gene called mecp2 which creates a
protein that is very important for brain
function
so first billions of copies at the mecp2
gene are manufactured in a lab then the
manufactured genes are packaged inside
the vectors in the upcoming clinical
trial a person with Brett will receive a
single injection of gene therapy product
called AVXS 201 into their cerebral
spinal fluid the vectors will then carry
their precious cargo to the brain and
deliver it into the nucleus of the cells
once this is accomplished the vector
naturally disintegrates and the new
mecp2 genes go to work making the
healthy and vital protein our hope is
that individuals will improve

English: 
dramatically what symptoms will improve
and by how much and in which individuals
these are questions that will remain
unanswered
until the clinical trial is undertaken
one thing we do know if improvements do
then they won't happen overnight it will
take time
intense therapies and lots of hard work
our SRT started funding gene therapy
experiments in 2010 and we've spent
almost 7 million dollars to date our
first gene therapy consortium launched
in 2014 and generated the data that led
to a vexes decision to pursue ret we're
extremely hopeful but we can't just sit
idle while we wait for trial results
which is why we launched gene therapy
consortium 2.0 last year to start
working on a second-generation gene
therapy product just in case the goals
of gene therapy 2.0 are to maximize the
number of brain cells the vector can
penetrate and also find ways to control

Chinese: 
不过到底什么症状能够改善，改善多少，并且对什么样的患者能够改善？
这些问题在临床试验没有开展前都无法回答。
不过有一点我们知道：如果有改善，这种改善也不会是一夜之间就发生。
它需要时间，大量的康复以及很多艰苦的工作。
RSRT从2010年开始资助基因疗法的实验，
迄今我们已经在这上面花费了7百万美元。
在2014年我们启动了第一个基因疗法联盟，
工作中获得的数据成果也让AveXis公司决心进军雷特治疗领域。
我们非常期望他们的成功，但是我们也不能干坐着等待临床试验结果。
这就是为什么去年我们启动了基因疗法联盟2.0，
开始为下一代基因疗法产品开展工作。以防万一嘛。
2.0的基因疗法的目标是让载体能够渗透到最大数量的大脑细胞中，

Chinese: 
同时寻找方法来控制新的健康基因制造的蛋白质的数量。
俗话说，射门多多益善，
所以我们同时也在资助其它一些有潜力的治愈方法，
包括DNA编辑、RNA编辑、RNA剪切、以及MECP2再激活项目。
看看吧，是你们的捐赠让这些变成了可能！
我们一起，会不遗余力的按照我们治愈路线图前进。
谢谢！

English: 
the amount of protein the new healthy
genes make the more shots on goal the
better which is why we're also funding a
number of other potentially curative
approaches including DNA editing RNA
editing RNA translating and mecp2
reactivation projects just look at what
your donations are making possible
together we will leave no stone unturned
on our roadmap to a cure thank you.
