Nature Medicine: New gene therapy fixes cause of hereditary deafness

Scientists at Fudan University in Shanghai, China have treated five children with a hereditary form of deafness using a new gene therapy. The respective research was published in the scientific journal Nature Medicine.

The children, from one to 11 years old, participated in the experiment. All had been deaf since birth due to inherited mutations in the OTOF gene, which disrupt the body's ability to produce the protein otoferlin, which transmits auditory signals from the ear to the brain. This pathology is associated with 2 to 8% of all congenital hearing disorders.

The research team used an inactive virus that delivered working copies of the affected gene to the inner ear. When the genetic material arrived at the desired location, the cells in the ear used it as "cues" to produce otoferlin.

As a result, all children began to hear in both ears 13 weeks after surgery.

According to scientists, gene therapy requires minimally invasive surgical intervention. However, using the virus as a carrier of the necessary genes is associated with the risk of an immune reaction in the body.

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