渐冻症的新疗法 分享到:
Genetic Repair for Disease that Turns a Body into a Prison 2018-04-1456552

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Stephen Hawking lived with ALS, a motor neuron disease, from the age of 21, as it gradually destroyed his body, leaving him unable to speak or move.Danny Baird wasn’t diagnosed with a motor neuron disease until she was much older.Her family is among the 10 percent of MND cases that are inherited. She watched her brothers and sisters die from the condition.
斯蒂芬·霍金从21岁开始就患有运动神经元疾病ALS,疾病逐渐摧毁了他的身体,使他无法说话或动弹。丹尼·贝尔德直到上了年纪才被诊断出患有运动神经元疾病,这是一种遗传性疾病,她的家族中患病人数占所有MND病例的百分之十,她目睹了自己兄弟姐妹因此而亡。
“That terrified me, because I’d seemed like, I knew what was ahead of me and so I’d seen and I was particularly close to my younger brother and I spent quite a lot of time with him when he had it, but never really thought that I’ll get it.”
这让我感到害怕,因为我觉得,我知道我的未来是什么,所以我知道的,当我弟弟得病的时候我和我的弟弟特别亲密,我花了很多的时间陪他,但是从来没有想到我会得病。
The Baird have a mutation in their genetic code, the sod1 gene, which affects the making of proteins. Doctors, using genetic technologies called anti sense oligonucleotides or ASO, are trying to stop the mutant gene from producing faulty and toxic protein.
贝尔德的基因编码中有一个突变,编码为sod1的基因会影响蛋白质的生成。医生们正试图使用一种被称为反义寡核苷酸或ASO的基因技术,阻止突变基因产生有缺陷和有毒的蛋白质。
“DNA sits in the nucleus and the protein machinery sits out in the cytoplasm. Every protein that’s made has to be read from the DNA.And a little transport mRNA it’s called is taken out and transported to the protein making machinery.It’s like the blueprint of every single protein that’s made now the proteins toxic.It’s very difficult for us to get access and correct the DNA at the moment, so we try and target this little transported RNA.The antisense oligonucleotides that we use which is the therapy is able to bind the RNA.An enzyme comes along RNAs and destroys the RNA which prevents the protein being made.”
DNA位于细胞核,而蛋白质机器位于细胞质中。每种蛋白质的产生都需要读取DNA,产出mRNA且运送到蛋白质生产机制中。这就像每个单一蛋白质的蓝图,而现在基因的错误导致有毒蛋白质的产生。现在我们很难获取并修正DNA,所以我们尝试修正这个小小的转运RNA。我们使用的反义寡核苷酸技术是一种能够结合RNA的治疗方法,这种产自RNA的酶会摧毁那些阻止蛋白质产生的RNA。
Sod1 is one of 13 genes which doctors believe can cause inherited motor neuron diseases.It’s believed to account for20 percent of such cases.Research is underway to learn more about the other genes, which may provide further clues about other forms of the disease.
医生认为sod1属于可导致遗传性运动神经元疾病的13种基因之一,这种情况占所有病例的百分之二十。目前正在对其他基因进行研究,这些基因可能会提供有关其他形式疾病的进一步线索。
I’m Faith Lapidus VOA news
我是VOA记者费思。

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