Innovative Gene Therapy Restores Hearing in Patients with Congenital Deafness
A novel gene therapy has successfully restored hearing in patients with genetic deafness, showing rapid and safe results in both children and adults, paving the way for personalized auditory treatments.
Recent advances in gene therapy have demonstrated promising results in restoring hearing for individuals with genetic forms of deafness or severe hearing impairments. A groundbreaking study led by researchers at Karolinska Institutet, published in Nature Medicine, involved 10 patients aged between 1 and 24 years from hospitals in China. These patients had mutations in the OTOF gene, which results in a deficiency of the otoferlin protein essential for transmitting auditory signals from the ear to the brain.
The therapy utilizes an adeno-associated virus (AAV) to deliver a healthy version of the OTOF gene directly into the inner ear through a minimally invasive injection at the cochlea's round window. The outcomes were remarkable, with all participants experiencing significant hearing improvements within just one month of treatment. Follow-up assessments over six months showed an average increase in perceivable sound volume from 106 decibels to 52 decibels.
Notably, younger patients, especially those aged five to eight, responded most positively, with cases like a seven-year-old girl who regained nearly complete hearing capacity and was able to communicate normally with her family after four months. This study is notable as it is the first to evaluate the efficacy of gene therapy in teenagers and adults with genetic deafness, expanding the potential scope of treatment.
Safety evaluations indicated that the procedure was well-tolerated. The most common side effect was a temporary reduction in neutrophils, a type of white blood cell, but no serious adverse effects were reported over the follow-up period of up to 12 months. Researchers highlight that this approach could be extended to other genetic causes of deafness, such as mutations in GJB2 and TMC1, which are more complex but have shown encouraging results in animal studies.
This advancement marks a significant step toward personalized genetic treatments for hearing loss, offering hope to many affected individuals and their families. Further research and larger clinical trials are ongoing to confirm the durability and broader application of this therapy.
Source: https://medicalxpress.com/news/2025-07-gene-therapy-deaf-patients.html
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