第2647期:Gene Treatment Lets Children Deaf at Birth Hear(2)

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.

研究人员说，一次性疗法在医疗操作过程中将该基因的工作副本赋予了内耳。 尽管两人的研究中的一个孩子在两个耳朵中都接受了治疗，但大多数孩子都被一只耳朵接受治疗。

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.

与六个孩子的研究在上海的福丹大学进行。 Yilai Shu博士帮助领导了这项研究，并接受了Chen的实验室的培训。 陈参与了研究。 中国科学组织和生物技术公司上海刷新的治疗学有助于提供财务支持。

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.

研究人员观察到孩子大约六个月。 他们不知道为什么治疗在其中一种中没有起作用。 研究人员说，但是五个完全聋哑的五个人现在可以听到正常的讨论。

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

陈估计他们现在听到的是正常水平的60％至70％。 该疗法没有引起重大副作用。

Early results from other research have shown similar results.

其他研究的早期结果显示出相似的结果。

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 Pharmaceuticals是一家位于纽约州的生物技术公司。 它在十月份宣布，两岁以下的儿童在基因治疗六周后表现出改善。 结果来自Regeneron在波士顿公司的Decibel Therapeutics的支持下进行的研究。

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.”

哥伦比亚大学的劳伦斯·卢斯蒂格（Lawrence Lustig）博士参与了Regeneron研究。 他说，尽管这些研究中的孩子们并没有听到很好的听力，但“即使这些孩子的听力损失恢复也令人震惊。”

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，仍然存在许多问题。 它们包括疗法将持续多长时间，以及儿童的听力是否会继续改善。

Some people question if gene therapy for deafness is ethical.

有些人质疑基因疗法是否对耳聋是道德的。

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.

Teresa Blankmeyer Burke是一位聋哑人，涉及医学道德的教授。 她在华盛顿特区的聋人大学加洛德大学（Gallaudet University）教授。

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，耳聋不会引起严重或致命的疾病。 布兰克迈尔·伯克（Blankmeyer Burke）说，与聋人社区成员合作有关基因疗法的重要性很重要。 她补充说，许多人认为基因疗法可能是对“签署聋人社区”的可能威胁。

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

但是，陈说：“这是表明基因疗法正在起作用的真正证据。” 他补充说：“这打开了整个领域。”