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双语新闻:中国研究人员发现:基因治疗可以让出生时失聪的孩子听到声音

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2024年01月30日

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

基因治疗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性疾病的医学治疗。


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

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岁男孩也有类似的改善。早些时候,中国研究人员发表了一项研究,显示另外两个孩子也有类似的改善。


The experimental methods, or therapies, target only one rare condition. But scientists say similar treatments could someday help many more children with other kinds of deafness caused by genes.

这些实验方法或疗法只针对一种罕见的疾病。但科学家表示,类似的治疗方法有一天可能会帮助更多由基因引起的其他类型耳聋的儿童。


Around the world, 34 million children have deafness or hearing loss. And information from the U.S. Centers for Disease Control and Prevention says genes are responsible for up to 60 percent of cases.

全世界有3400万儿童患有耳聋或听力损失。来自美国疾病控制和预防中心的信息称,基因导致了高达60%的病例。


Deafness caused by genes is passed down from parents to children. It is called hereditary deafness. It is the latest condition scientists are targeting with gene therapy. Gene therapy is already approved to treat illnesses such as sickle cell disease and severe hemophilia.

由基因引起的耳聋是由父母遗传给孩子的。这被称为遗传性耳聋。这是科学家们用基因疗法治疗的最新目标。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叫做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. He is a writer of the study published in The Lancet. "We couldn't be more happy or excited about the results."

“没有任何治疗可以逆转听力损失……这就是为什么我们一直在努力开发一种治疗方法,”波士顿大众眼耳治疗中心的陈正毅说。他是发表在《柳叶刀》上的这项研究的作者之一。“我们对结果感到非常高兴和兴奋。”


Treatment results

治疗结果


The team recorded videos of patients. One shows a baby, who formerly could not hear at all, looking back in response to a doctor's words six weeks after treatment. Another shows a little girl 13 weeks after treatment repeating father, mother, grandmother, sister, and "I love you."

该团队录制了患者的视频。其中一幅画的是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。另一张照片显示,一个小女孩在接受治疗13周后重复着父亲、母亲、祖母、妹妹和“我爱你”。


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

所有参与实验的儿童都患有一种被认为会导致2%到8%的遗传性耳聋的疾病。它是由一种负责内耳蛋白质的基因发生变化引起的,这种蛋白质被称为耳蛋白,它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. Most of the children were treated in one ear, although one child in the two-person study was treated in both ears.

研究人员表示,这种一次性疗法可以在医疗手术中将该基因的工作副本植入内耳。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. Dr. Yilai Shu helped lead the study and trained in Chen's laboratory. Chen was involved in the research. Chinese science organizations and biotechnology company Shanghai Refreshgene Therapeutics helped provide financial support.

该研究在上海复旦大学对6名儿童进行。舒易来博士帮助领导了这项研究,并在陈的实验室接受了培训。陈也参与了这项研究。中国的科学组织和生物技术公司上海清新生物技术有限公司提供了资金支持。


Researchers observed the children for about six months. They do not know why the treatment did not work in one of them. But the five others, who were completely deaf, can now hear a normal discussion, the researchers said.

研究人员对这些孩子进行了大约六个月的观察。他们不知道为什么治疗在其中一人身上不起作用。但研究人员说,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. The therapy caused no major side effects.

陈估计他们现在的听力水平是正常水平的60%到70%。这种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. It announced in October that a child under two years old showed improvements six weeks after gene therapy. The results came from a study Regeneron did with support from Decibel Therapeutics, a company in Boston.

Regeneron制药公司是一家总部位于纽约州的生物技术公司。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病情有所改善。结果来自Regeneron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. 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."

哥伦比亚大学的Lawrence Lustig博士参与了Regeneron的研究。他说,尽管这些研究中的孩子最终并没有得到很好的听力,“即使是这些孩子听力损失的中度恢复也是相当惊人的。”


He added that many questions remain. They include how long the therapies will last and if hearing will continue to improve in the children.

他补充说,还有许多问题有待解决。它们包括治疗将持续多久,以及儿童的听力是否会继续改善。


Some people question if gene therapy for deafness is ethical.

一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. She teaches at Gallaudet University, a university for deaf people in Washington, D.C. She said that there is no agreement about the need for gene therapy targeting deafness.

特蕾莎·布兰克梅尔·伯克是一位教授,她是聋人,专门研究医学伦理。她在加劳德特大学任教,这是一所位于华盛顿特区的聋人大学。她说,目前还没有就针对耳聋的基因治疗的必要性达成一致。


She also pointed out that deafness does not cause severe or deadly illness. Blankmeyer Burke said that it is important to work with deaf community members about the importance of gene therapy. She added gene therapy is seen by many as a possible threat to "signing Deaf communities."

她还指出,耳聋不会导致严重或致命的疾病。布兰克迈尔·伯克说,与聋人社区成员一起了解基因治疗的重要性是很重要的。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." And he added, "It opens up the whole field."

然而,陈教授说:“这是证明基因疗法有效的真正证据。”他补充说,“它打开了整个领域。”



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