Skip to main content

Gene Therapy Restores Hearing in Deaf Mice

A new gene therapy tested on deaf mice proved to partially restore hearing and balance, giving hope to many who suffer from Usher syndrome, a form of human deafness that usually comes with blindness. 

While the research, which was published in the journal Natural Medicine, is still in its early stages and has not been tested on humans, it has made many hopeful of a future therapy for the deaf. 

Michelle Hastings, lead researcher, focused in on a gene called USH1C, which is "Type 1" of Usher syndrome.

The role of the gene is to produce a protein called harmonin, which plays an important role in hair cells. Our hearing is processed in the cochlea of the inner ear, where these hair cells are located, and an electrical signal is sent to the brain.

The team created a strand of genetic material called an antisense oligonucleotide to "switch off" a faulty gene that truncates forms of harmonin, leading to deafness. 

Once this therapy was inserted into mice that were born with the mutation, their hearing was restored at low frequency. It also reduced head tossing, a behavior that occurs when balance is impaired. 

"These effects were sustained for several months, providing evidence that congenital deafness can be effectively overcome by treatment early in development to correct gene expression," the study said. 

After the experiment concluded, the mice were dissected and researchers found their cochleas to have grown some hair cells. 

Much progress has been made recently in researching potential treatments for the deaf.

In January, doctors at the Massachusetts Eye and Ear and Harvard Medical School reported on another gene drug that transformed cochlea cells into hair cells. 

Last year, a faulty version of a gene called VGLUT3 was targeted and fixed. The gene also controls a protein vital for hair cells to send signals.



Popular Video