Chinese researchers cure deafness with genetic treatment

They used a harmless virus to address the otoferlin gene defect in children.

In a remarkable scientific advancement, Chinese researchers have reported a groundbreaking genetic treatment that has enabled deaf children to gain the gift of hearing.

According to a MIT Technology Review report, this pioneering experiment involved the modification of a benign virus by introducing new genetic information. Subsequently, the engineered virus was injected into the cochlea of the children participating in the experiment, a coiled, fluid-filled cavity within the ear. 

The results obtained by Fudan University in Shanghai during this research indicate that hearing was restored in four out of five children who received the treatment.

The genetic material incorporated into the virus was a replica of the otoferlin gene. This gene encodes a protein with the same name, crucial for transmitting auditory information from the inner ear to the brain.

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The new treatment will not help everyone who is deaf.

It applies only to one specific cause of deafness at birth: a defect in a gene that produces a protein called otoferlin. The inner ear contains about 16,000 hair cells, so called because they have comblike extensions that vibrate to different frequencies of sound. Without otoferlin, these cells can’t transmit the chemicals that relay information to the brain.

Approximately 1 to 3 percent of congenital deafness cases worldwide can be attributed to defects in the otoferlin gene.

When such genetically modified viruses were introduced into the ear, they effectively replaced the faulty gene, providing the children with approximately 60 to 65 percent of the typical hearing capacity.

The new treatment is designed to add a working copy of the otoferlin gene. Because of the gene’s large size—it is around 6,000 DNA letters long—it had to be broken into two parts, each packaged separately into millions of copies of a harmless virus. Shu then carefully injects the loaded viruses deep into a fluid-filled chamber in a part of the children’s ears called the cochlea.

Once inside the body, the two sections of DNA recombine to make a complete gene able to guide the production of the missing otoferlin protein, MIT Technology Review quoted the authors of this study as saying.

Although this treatment specifically targets a rare form of deafness, scientists believe that it could serve as a stepping stone for restoring hearing in cases of other congenital deafness.

Researchers outside China are also actively engaged in developing genetic therapies targeting the otoferlin gene. For instance, projects at Cambridge University and Regeneron, a pharmaceutical company in the United States, have shown promising results too.

Last October, Regeneron announced positive outcomes from a clinical trial involving a child who had undergone a pretty-much similar genetic therapy treatment. 


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