A new gene therapy trial has helped 10 children and young adults with congenital deafness to hear for the first time in their lives. All 10 patients, ages one to 24, gained some hearing ability after treatment. The therapy targets a gene called OTOF, which affects about 200,000 people worldwide. When this gene doesn't work properly, it stops sound signals from traveling from the ear to the brain.
Doctors used a special virus to deliver a healthy copy of the gene directly into the inner ear through one injection. The treatment worked best for younger patients, especially those between 5 and 8 years old. One 7-year-old girl recovered almost all her hearing within four months and could talk normally with her mother. After treatment, she heard rain falling for the first time when leaving the doctor's office.
The average sound level patients could hear improved from 106 decibels to 52 decibels after six months. Researchers will follow these patients for 5-10 years to see how long the treatment lasts.
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