Breakthrough in gene therapy restores hearing in children with genetic deafness

A groundbreaking development in gene therapy has given hope to children with genetic hearing disabilities. This achievement is a major step forward in restoring hearing for those who couldn’t hear due to inherited hearing impairments. Genetic hearing loss makes up about 50% of all cases of hearing impairment, while the remaining cases result from factors like infections or physical trauma. Scientists in China made this breakthrough and unveiled it at a meeting in Belgium on November 1.

In China, four children may now be the first in the world to have their natural hearing abilities restored through gene therapy. This accomplishment is remarkable because, until now, there hasn’t been any drug or treatment capable of significantly improving hearing. According to Yilai Shu, a surgeon and scientist at Fudan University in Shanghai who led the experiment, the team proceeded with caution, aware of the groundbreaking nature of their work.

The treatments began in December, and the results have been promising. One child, who had never spoken before, has now started to say “baba” and “mama” after the treatment. This is an emotional moment for the parents, as it marks a significant improvement in their child’s quality of life. Before the treatment, these children couldn’t hear even the loudest sounds in a movie theater. However, they can now hear close to normal speech, and one can even hear a whisper.

So, how did this remarkable achievement come about? These children were born with hearing disabilities because they had two defective copies of a gene responsible for a protein that helps the inner ear’s hair cells transmit sound to the brain. To address this, researchers used harmless viruses to introduce a functional copy of the otoferlin gene into the children’s ears. The result was that four out of five treated patients now have some degree of hearing improvement.

A video shared by the MIT Technology Review shows a heartwarming moment where a 6-year-old child is chatting with her mother after her cochlear implant was removed. This demonstrates the tangible impact of the treatment on the child’s life.

It’s important to note that the specific hearing disability related to the otoferlin gene is quite rare, accounting for only 1 to 3 percent of inherited deafness cases. However, the success of this gene therapy offers hope for treating other genetic forms of hearing loss, which may impact a larger portion of the population. This breakthrough in gene therapy is a beacon of hope for individuals who have been living with hearing disabilities, and it represents a significant leap in the field of medical science.

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