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基因疗法让遗传性耳聋患者听到声音

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Gene treatments are being credited with permitting several children born with deafness to hear again.

基因疗法使几个天生失聪的孩子重获听力。

Gene therapy is a medical treatment that aims to change a few of a patient's genes to cure a genetic disease.

基因疗法是一种旨在改变患者的一些基因以治愈遗传病的医学疗法。

A small study recently published in The Lancet reported improvements in the hearing in five of six children who were treated in China.

最近发表在《柳叶刀》上的一项小型研究报告称,在中国接受治疗的六名儿童中,有五名的听力有所改善。

Around the same time, the Children's Hospital of Philadelphia in the U.S. eastern state of Pennsylvania announced similar improvements in an 11-year-old boy treated there.

大约在同一时间,美国东部宾夕法尼亚州的费城儿童医院宣布,在那里接受治疗的一名11岁男孩的听力也有所改善。

And earlier, Chinese researchers published a study showing similar improvements in two other children.

早些时候,中国研究人员发表的一篇研究论文称,另外两个孩子的听力有所改善。

The experimental methods, or therapies, target only one rare condition.

这种实验方法只针对一种罕见的疾病。

But scientists say similar treatments could someday help many more children with other kinds of deafness caused by genes.

但科学家表示,类似的治疗方法有朝一日可能会帮助更多患有其他类型由基因引起的耳聋的儿童。

Around the world, 34 million children have deafness or hearing loss.

全世界有3400万儿童患有耳聋(即听力损失)。

And information from the U.S. Centers for Disease Control and Prevention says genes are responsible for up to 60 percent of cases.

来自美国疾病控制与预防中心的信息称,高达60%的病例与基因有关。

Deafness caused by genes is passed down from parents to children.

由基因引起的耳聋是由父母遗传给孩子的。

It is called hereditary deafness.

这被称为遗传性耳聋。

It is the latest condition scientists are targeting with gene therapy.

这是科学家用基因疗法治疗的最新疾病。

Gene therapy is already approved to treat illnesses such as sickle cell disease and severe hemophilia.

基因疗法已经被批准用于治疗镰状细胞病和严重血友病等疾病。

Children with hereditary deafness often get a device called a cochlear implant that helps them hear sound.

患有遗传性耳聋的儿童通常会植入一种名为人工耳蜗的设备来帮助他们听到声音。

"No treatment could reverse hearing loss…That's why we were always trying to develop a therapy," said Zheng-Yi Chen of Boston's Mass Eye and Ear, a treatment center for eye and ear problems.

波士顿大众眼耳治疗中心(一家治疗眼耳疾病的治疗中心)的陈正毅(音译)说:“没有任何治疗方法可以逆转听力损失,这就是我们一直在努力开发一种治疗方法的原因。”

He is a writer of the study published in The Lancet.

他是发表在《柳叶刀》上的这篇研究论文的作者之一。

"We couldn't be more happy or excited about the results."

“我们对结果感到非常高兴和兴奋。”

The team recorded videos of patients.

该团队录制了患者的视频。

One shows a baby, who formerly could not hear at all, looking back in response to a doctor's words six weeks after treatment.

其中一段视频显示了一个以前完全听不见声音的婴儿在接受治疗六周后在医生的指导下开始回头看。

Another shows a little girl 13 weeks after treatment repeating father, mother, grandmother, sister, and "I love you."

另一段视频显示,一个小女孩在接受治疗13周后重复着父亲、母亲、祖母、姐妹和“我爱你”。

All the children in the experiments have a condition that is believed to cause two to eight percent of hereditary deafness.

所有参与实验的儿童都患有一种据信会导致2%至8%的遗传性耳聋的疾病。

It is caused by changes in a gene responsible for an inner ear protein called otoferlin, which helps hair cells send sound signals to the brain.

它是由一种控制名为耳畸蛋白的内耳蛋白的基因发生变化引起的,这种蛋白帮助毛细胞向大脑发送声音信号。

The researchers say that the one-time therapy gives a working copy of that gene to the inner ear during a medical operation.

研究人员表示,这种一次性疗法在医疗手术期间将该基因的工作副本植入内耳。

Most of the children were treated in one ear, although one child in the two-person study was treated in both ears.

大多数儿童接受了单耳治疗,尽管在两人研究中有一名儿童接受了双耳治疗。

The study with six children took place at Fudan University in Shanghai.

这项针对六个孩子的研究是在上海复旦大学进行的。

Dr. Yilai Shu helped lead the study and trained in Chen's laboratory.

舒亦来医生帮助领导了这项研究,并在陈的实验室接受了培训。

Chen was involved in the research.

陈参与了这项研究。

Chinese science organizations and biotechnology company Shanghai Refreshgene Therapeutics helped provide financial support.

中国的科学组织和生物技术公司上海清新基因治疗公司提供了资金支持。

Researchers observed the children for about six months.

研究人员对这些孩子进行了大约六个月的观察。

They do not know why the treatment did not work in one of them.

他们不知道该疗法在其中一人身上不起作用的原因。

But the five others, who were completely deaf, can now hear a normal discussion, the researchers said.

但研究人员表示,另外五名完全失聪的人现在可以听到正常的谈话了。

Chen estimated they now hear at a level 60 percent to 70 percent of normal.

陈估计,他们现在的听力水平是正常水平的60%到70%。

The therapy caused no major side effects.

该疗法没有产生严重的副作用。

Early results from other research have shown similar results.

其他研究的早期结果也显示了类似的结果。

Regeneron Pharmaceuticals is a biotech company based in New York state.

再生元制药公司是一家总部位于纽约州的生物技术公司。

It announced in October that a child under two years old showed improvements six weeks after gene therapy.

该公司在10月份宣布,一名两岁以下的儿童在接受基因治疗六周后听力有所改善。

The results came from a study Regeneron did with support from Decibel Therapeutics, a company in Boston.

这一结果来自再生元公司在波士顿分贝治疗公司的支持下进行的一项研究。

Columbia University's Dr. Lawrence Lustig is involved in the Regeneron study.

哥伦比亚大学的劳伦斯·拉斯帝格博士参与了再生元公司的研究。

He said although the children in these studies do not end up with very good hearing, "even a moderate hearing loss recovery in these kids is pretty astounding."

他说,尽管这些研究中的孩子最终的听力并不是很好,“即使这些孩子的听力轻度恢复,也是相当令人震惊的。”

He added that many questions remain.

他补充说,还有许多问题有待解决。

They include how long the therapies will last and if hearing will continue to improve in the children.

其中包括治疗将持续多长时间,以及儿童的听力是否会继续改善。

Some people question if gene therapy for deafness is ethical.

有些人质疑基因治疗耳聋是否合乎伦理。

Teresa Blankmeyer Burke is a professor who is deaf and who deals with medical ethics.

特蕾莎·布兰克迈尔·伯克是一名聋人教授,专门研究医学伦理。

She teaches at Gallaudet University, a university for deaf people in Washington, D.C.

她在加拉德特大学任教,这是一所位于华盛顿特区的聋人大学。

She said that there is no agreement about the need for gene therapy targeting deafness.

她说,对于针对耳聋的基因治疗的必要性,目前还没有达成一致意见。

She also pointed out that deafness does not cause severe or deadly illness.

她还指出,耳聋不会导致严重或致命的疾病。

Blankmeyer Burke said that it is important to work with deaf community members about the importance of gene therapy.

布兰克迈尔·伯克表示,与聋人社区成员一起了解基因治疗的重要性是很重要的。

She added gene therapy is seen by many as a possible threat to "signing Deaf communities."

她补充说,基因疗法被许多人视为对“手语聋人社区”的潜在威胁。

However, Chen said: "This is real proof showing gene therapy is working."

然而,陈说:“这是证明基因疗法有效的真正证据。”

And he added, "It opens up the whole field."

他补充说,“它打开了整个领域的大门。”

I'm Faith Pirlo. And I'm Gregory Stachel.

费斯·皮尔洛、格雷戈里·施塔赫尔为您共同播报。

译文为可可英语翻译,未经授权请勿转载!

重点单词   查看全部解释    
hemophilia [.hi:mə'filiə]

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n. 血友病

联想记忆
announced [ə'naunst]

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宣布的

 
recovery [ri'kʌvəri]

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n. 恢复,复原,痊愈

 
ethical ['eθikəl]

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adj. 道德的,伦理的,民族的

 
therapy ['θerəpi]

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n. 疗法,治疗

 
threat [θret]

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n. 威胁,凶兆
vt. 威胁, 恐吓

 
community [kə'mju:niti]

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n. 社区,社会,团体,共同体,公众,[生]群落

联想记忆
lancet ['lɑ:nsit]

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n. [医]柳叶刀,[建]尖顶窗

联想记忆
reverse [ri'və:s]

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n. 相反,背面,失败,倒档
adj. 反面的

联想记忆
hereditary [hi'reditəri]

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adj. 世袭的,遗传的

 

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