In this project, Dr Erik de Vrieze at Radboud University Medical Center, Netherlands, develops a genetic therapy for a common form of inherited, adult-onset hearing loss.
Project start date: October 2022
Project end date: March 2027
About the project
While hearing loss in adults is more often caused by factors such as noise exposure or ageing, genetic factors can also contribute. A common type of adult-onset hearing loss, called DFNA9, is due to mutations in the COCH (cochlin) gene.
The mutated gene leads to the production of toxic cochlin proteins. In this project, researchers explore potential treatments that block the production of these toxic cochlin proteins. This should also protect the hearing of people at risk of this genetic form of hearing loss or prevent worsening of their hearing loss, especially if the treatment was given at an early age, before hearing loss begins to develop.
How it works
The researchers have created special molecules called antisense oligonucleotides (ASOs). These ASOs specifically target the mutated version of the COCH gene and prevent it from being used as a blueprint to make proteins. This way, the ASOs specifically block production of the toxic cochlin protein.
Using these ASOs in the inner ear could slow or even stop the progression of hearing loss in people with DFNA9. In this project, the researchers will conduct experiments to show this approach is effective and safe for use in the inner ear. Their overall aim is to develop a new genetic therapy to prevent hearing loss in an adult-onset form of hereditary deafness, DFNA9.
How will this research benefit people at risk of hearing loss?
People with DFNA9 hearing loss typically start to lose their hearing as young to middle-aged adults and it worsens with time. They may also develop problems with their balance. The results from this project will provide important data to support future studies of this new genetic therapy towards approval for testing in clinical trials. If successful, their work will advance a new genetic treatment for DFNA9 hearing loss.
This will open the way for this promising approach to be adapted to treat other forms of inherited hearing loss and prevent hearing loss in large numbers of people.
About the researcher
Dr Erik de Vrieze is Assistant Professor at Radboud University Medical Center, Netherlands. He was awarded this RNID-FPA Translational Research Grant in 2021, which is funded in partnership with Fondation Pour l’Audition.
I hope that I can offer treatments to several large groups of people who have a form of inherited hearing loss that means they will lose their hearing at some point in their life, a form of early-onset adult hearing loss. Many of them have seen the effects of this in their family members. If treated early, the progression or development of hearing loss could be stopped or delayed in the younger generations of these families.”
