Genetic Treatment Successfully Restores Hearing in Individuals Struggling with Hearing Impairment
A groundbreaking study, published in *Nature Medicine*, has shown that gene therapy can successfully restore hearing in individuals with congenital deafness caused by mutations in the OTOF gene. The trial, conducted at five sites in China, involved ten participants aged between 1.5 to 23.9 years and demonstrated significant improvements in hearing within a month of treatment.
The study, which is ongoing, has shown that the treatment is safe and well-tolerated, with no serious adverse events reported during a 6-12 month follow-up period. The treatment involves using a synthetic adeno-associated virus (AAV) to deliver a functional version of the OTOF gene to the inner ear.
Key findings from the study include rapid improvement in hearing, with most patients showing improvements within a month, and all patients experiencing significant improvement by six months. The treatment was particularly effective in younger patients, with optimal outcomes observed in 5- to 8-year-olds.
Researchers view this as a significant step in the genetic treatment of deafness, offering hope for life-changing outcomes for both children and adults with congenital deafness due to OTOF gene mutations. The study's primary endpoints were safety and tolerability within 5 years, and secondary endpoints assessed auditory function.
Post hoc analyses showed that the therapeutic effect was rapid, taking 1 month to achieve most of the overall hearing improvement. The average click auditory brainstem response (ABR) threshold, the tone-burst ABR threshold, and the auditory steady-state response also improved.
As the trial continues, researchers are expanding their work to other genes that cause deafness such as GJB2 and TMC1. The trial's registration number is NCT05901480. The study was conducted in collaboration with hospitals and universities in China.
This latest progress in gene therapy for congenital deafness marks the first time older age groups, including teenagers and adults, have been successfully treated with this method. The treatment offers a promising solution for individuals living with congenital deafness, providing hope for improved hearing and a better quality of life.
- The groundbreaking gene therapy study, which aims to restore hearing in individuals with congenital deafness caused by OTOF gene mutations, also plans to explore other genes that cause deafness like GJB2 and TMC1.
- The treatment for congenital deafness, currently being studied, uses a synthetic adeno-associated virus (AAV) to deliver a functional version of the OTOF gene to the inner ear, potentially benefiting medical-conditions related to the brain and health-and-wellness.
- Neuroscience news outlets are reporting significant improvements in hearing within a month of treatment for patients with congenital deafness, following the successful trial of a gene therapy, with all patients experiencing further improvement by six months.
- As the study on gene therapy for congenital deafness continues, its primary endpoints are focused on safety and tolerability within 5 years, while secondary endpoints assess auditory function, including factors such as the average click auditory brainstem response (ABR) threshold, the tone-burst ABR threshold, and the auditory steady-state response.
