A new gene therapy is bringing hope to children born with a specific type of genetic deafness. This year, researchers at China’s Fudan University and Harvard Medical School (HMS) announced that the groundbreaking treatment had 1 the hearing of five of the six test patients.
The study was 2 six children between the ages of one and seven. Of the six, four had cochlear implants (人工耳蜗). They allowed them 3 in one ear. When the implants were 4 , all participants were completely deaf. The two youngest 5 , ages one and two, had no implants.
The 6 children were born with a rare genetic disability. For the lack of the 7 OTOF gene, they cannot hear things. In the trial, the children were all injected in the cochlea (耳蜗) with two harmless viruses. 8 virus contained half of the normal gene. 9 the gene reached the target cells, it began to work. Within four to six weeks after the 10 , the researchers began testing for signs of hearing improvement.
At the end of the 26 weeks, five of the six children showed substantial 11 in hearing. The three older children could 12 and respond to speech with their implants turned off. Two could 13 recognize speech in a noisy room and 14 a phone conversation. The youngest children also showed improvement in their 15 to recognize speech. HMS Associate Professor Zheng-Yi Chen, who co-led the study, said that the one-year-old began to respond to sounds and 16 simple first words like “mama.” The team is 17 why one of the participants showed no improvement in hearing. They suspect the child’s body may have 18 the viruses.
More 19 are needed before the treatment can be offered to everyone. But once ready, it will 20 the lives of the estimated 200,000 people worldwide.
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