The researchers tested the treatment on lab mice. They did so by injecting a virus that carried a healthy gene right into their inner years.
Mice which suffered from deafness fully recovered their hearing after they benefited from treatment with a TMC1 gene.
The TMC1 gene is responsible for 4 to 8 percent of deafness cases. It role in hearing is of utmost importance because it codes for very important inner ear protein.
The researchers believe their findings are very important for future treatment because there are many children who will have their hearing restored.
Therefore, those that are born with defective genes could have a chance to a better life, where impaired hearing is not a problem and they can benefit from formal education like other children, without needing constant supervision.
“I can envision patients with deafness having their genome sequenced and a tailored, precision medicine treatment injected into their ears to restore hearing,” said lead study author, Dr. Jeffrey Holt, who works at Boston’s Children’s Hospital. The researchers are planning to implement the new treatment in 5 to 10 years from now.
There are more than 70 defective genes that can lead to deafness. This particular treatment only referred to TMC1, and it was tested on two forms of deafness related to this gene in humans. While one of them did not have a functional TMC1 gene, the other one had an unusual type of TMC1 gene.
Dr Holt explained that children who suffer from the former condition become deaf in their first years of life, while those who suffer from the latter go deaf later in life, when they are 10 to 15 year old.
The mice in the first group were treated with gene therapy and the other ones, belonging to the second group benefitted from treatment with a related gene, called TMC 2. The second group of mice only had a partial restoration of their hearing but the treatment was considered a success at the brain level.
0.3 percent of the newly born children will be affected by deafness. Even if there are cochlear implants and hearing aids, they are often not very effective, so this new treatment will be welcome by many specialists in the field, as soon as it becomes available.
A report of the research was published in the journal Science Translational Medicine.
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