VOA健康报道2024--Studies: Gene Treatment Lets Children Deaf at Birth Hear

Studies: Gene¹ Treatment Lets Children Deaf at Birth Hear

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

Gene therapy is a medical treatment that aims to change a few of a patient's genes² to cure a genetic³ disease.

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

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.

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.

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.

Deafness caused by genes is passed down from parents to children. It is called hereditary⁴ deafness. It is the latest condition scientists are targeting with gene therapy. Gene therapy is already approved to treat illnesses such as sickle⁵ cell disease and severe hemophilia.

Children with hereditary deafness often get a device called a cochlear implant⁶ that helps them hear sound.

"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."

Treatment results

The team recorded videos of patients. One shows a baby, who formerly⁷ 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."

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.

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.

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.

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.

Chen estimated they now hear at a level 60 percent to 70 percent of normal. The therapy caused no major side effects.

Early results from other research have shown similar results.

Regeneron Pharmaceuticals⁸ 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 Decibel⁹ Therapeutics, a company in Boston.

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 astounding¹⁰."

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

Some people question if gene therapy for deafness is ethical¹¹.

Teresa Blankmeyer Burke is a professor who is deaf and who deals with medical ethics¹². 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.

She also pointed¹³ 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."

However, Chen said: "This is real proof showing gene therapy is working." And he added, "It opens up the whole field."

Words in This Story

hereditary –adj. passed down from one generation to the next

implant –n. (medical) something connected to a person's body through a medical operation

reverse – v. to change (something) to an opposite state or condition

excited – adj. very enthusiastic and eager about something

pretty – adv. to a great degree or extent: very

astounding – adj. causing a feeling of great surprise or wonder

ethical – adj. following accepted rules of behavior: morally right and good

1 gene   

n.遗传因子，基因

参考例句：

• A single gene may have many effects.单一基因可能具有很多种效应。
• The targeting of gene therapy has been paid close attention.其中基因治疗的靶向性是值得密切关注的问题之一。

2 genes   

n.基因( gene的名词复数 )

参考例句：

• You have good genes from your parents, so you should live a long time. 你从父母那儿获得优良的基因，所以能够活得很长。 来自《简明英汉词典》
• Differences will help to reveal the functions of the genes. 它们间的差异将会帮助我们揭开基因多种功能。 来自英汉非文学 - 生命科学 - 生物技术的世纪

3 genetic   

adj.遗传的，遗传学的

参考例句：

• It's very difficult to treat genetic diseases.遗传性疾病治疗起来很困难。
• Each daughter cell can receive a full complement of the genetic information.每个子细胞可以收到遗传信息的一个完全补偿物。

4 hereditary   

adj.遗传的，遗传性的，可继承的，世袭的

参考例句：

• The Queen of England is a hereditary ruler.英国女王是世袭的统治者。
• In men,hair loss is hereditary.男性脱发属于遗传。

5 sickle   

n.镰刀

参考例句：

• The gardener was swishing off the tops of weeds with a sickle.园丁正在用镰刀嗖嗖地割掉杂草的顶端。
• There is a picture of the sickle on the flag. 旗帜上有镰刀的图案。

6 implant   

vt.注入，植入，灌输

参考例句：

• A good teacher should implant high ideals in children.好教师应该把高尚理想灌输给孩子们。
• The operation to implant the artificial heart took two hours.人工心脏植入手术花费了两小时。

7 formerly   

adv.从前，以前

参考例句：

• We now enjoy these comforts of which formerly we had only heard.我们现在享受到了过去只是听说过的那些舒适条件。
• This boat was formerly used on the rivers of China.这船从前航行在中国内河里。

8 pharmaceuticals   

n.医药品；药物( pharmaceutical的名词复数 )

参考例句：

• the development of new pharmaceuticals 新药的开发
• The companies are pouring trillions of yen into biotechnology research,especially for pharmaceuticals and new seeds. 这些公司将大量资金投入生物工艺学研究，尤其是药品和新种子方面。 来自《简明英汉词典》

9 decibel   

n.分贝(音量的单位)

参考例句：

• The noise pollution reached a high decibel level.噪音污染达到了很高分贝。
• In sound we use the decibel scale.在声学中我们采用分贝标度。

10 astounding   

adj.使人震惊的vt.使震惊，使大吃一惊astound的现在分词)

参考例句：

• There was an astounding 20% increase in sales. 销售量惊人地增加了20%。
• The Chairman's remarks were so astounding that the audience listened to him with bated breath. 主席说的话令人吃惊，所以听众都屏息听他说。 来自《简明英汉词典》

11 ethical   

adj.伦理的，道德的，合乎道德的

参考例句：

• It is necessary to get the youth to have a high ethical concept.必须使青年具有高度的道德观念。
• It was a debate which aroused fervent ethical arguments.那是一场引发强烈的伦理道德争论的辩论。

12 ethics   

n.伦理学；伦理观，道德标准

参考例句：

• The ethics of his profession don't permit him to do that.他的职业道德不允许他那样做。
• Personal ethics and professional ethics sometimes conflict.个人道德和职业道德有时会相互抵触。

13 pointed   

adj.尖的，直截了当的

参考例句：

• He gave me a very sharp pointed pencil.他给我一支削得非常尖的铅笔。
• She wished to show Mrs.John Dashwood by this pointed invitation to her brother.她想通过对达茨伍德夫人提出直截了当的邀请向她的哥哥表示出来。