Gene therapy successfully restores hearing in deaf patients
In a groundbreaking medical advancement, gene therapy has successfully restored hearing in deaf patients, offering new hope for those with genetic forms of hearing loss. The treatment works by delivering functional genes to the inner ear, repairing damaged or missing auditory functions. This marks a significant step forward in precision medicine and demonstrates the potential of gene therapy to reverse certain congenital conditions, paving the way for broader applications in hearing restoration and beyond.
A groundbreaking study involving Karolinska Institutet in Sweden has revealed that gene therapy can significantly restore hearing in both children and adults suffering from congenital deafness or severe hearing loss. All ten patients in the trial showed notable improvement, and the therapy was well-tolerated.
“This is a huge step forward in the genetic treatment of deafness, one that can be life-changing for children and adults,” said Dr. Maoli Duan, consultant and docent at the Department of Clinical Science, Intervention and Technology, Karolinska Institutet.
Study Overview
Conducted in collaboration with hospitals and universities in China
Published in the prestigious journal Nature Medicine
Included 10 participants aged between 1 and 24 years
All participants had mutations in the OTOF gene, which is responsible for a protein called otoferlin
Why the OTOF Gene Matters
Otoferlin plays a vital role in transmitting sound signals from the inner ear to the brain
Mutations in the OTOF gene cause a lack of otoferlin, leading to hearing impairment or deafness
How the Therapy Works
A synthetic adeno-associated virus (AAV) was used to deliver a working version of the OTOF gene
The injection was delivered through the round window of the cochlea (a membrane at the base of the inner ear)
The treatment involved only a single injection
Promising Results
Rapid effects: Most patients showed hearing recovery within a month
Six-month follow-up showed:
Improved hearing in all participants
Average hearing threshold improved from 106 dB to 52 dB
Children aged 5 to 8 responded the best
A 7-year-old girl regained almost full hearing and was having normal conversations with her mother just four months after the procedure
Breakthroughs for All Ages
This is the first study to show successful results not only in children but also in teenagers and adults
Several adult patients experienced significant hearing restoration
“Smaller studies in China have previously shown positive results in children, but this is the first time that the method has been tested in teenagers and adults too,” noted Dr. Duan.
Safety and Tolerability
The therapy was safe and well-tolerated
The most common side effect was a temporary drop in neutrophil levels (a type of white blood cell)
No serious adverse events were reported during 6 to 12 months of follow-up
The Future of Hearing Treatment
OTOF is just the beginning, says Dr. Duan
Ongoing research now focuses on other genes linked to deafness:
GJB2
TMC1
These genes are more complex to treat, but animal studies are showing promising early results
Global Collaboration
The research was conducted with partners including:
Zhongda Hospital and Southeast University in China
Funding came from:
Chinese research programs
Otovia Therapeutics Inc., which also developed the therapy and employs many involved researchers
Conclusion
This remarkable achievement represents a revolutionary advance in genetic medicine. For patients and families affected by hereditary hearing loss, gene therapy offers new hope. As science progresses, therapies like this could transform the lives of countless individuals worldwide, potentially curing various forms of genetic deafness.
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