A treatment developed by Israeli and US researchers has preserved inner ear hair cells in animal studies, pointing toward future options for conditions that currently have no cure
A collaboration between Tel Aviv University and US research teams has produced a gene therapy that protects the inner ear’s sensory hair cells from degeneration.
The approach, which targets mutations in the CLIC5 gene, could potentially reshape treatment strategies for inherited hearing loss and balance disorders if it proves successful in human trials.
Hundreds of millions affected
Hearing loss affects more than 430 million people worldwide and is projected to rise sharply by 2050. Roughly half of congenital cases stem from genetic mutations.
Balance disorders rooted in inner ear dysfunction affect up to one in five adults and increase the risk of falls.
Current treatments improve perception but do not prevent progressive cell loss. A therapy that intervenes at the genetic level could change long-term outcomes for those with genetic hearing loss.
The science behind the therapy
The CLIC5 gene supports the structural stability of the hair cells that enable hearing and balance. When the gene is faulty these cells deteriorate, leading first to hearing loss and then to balance problems.
The new therapy uses a self-complementary adeno-associated virus to deliver a healthy copy of the gene more efficiently and at lower doses than conventional vectors.
In animal models this technique prevented hair cell degeneration and preserved both auditory and vestibular function.
The work led by Professor Karen Avraham of Tel Aviv University with collaborators at Boston Children’s Hospital and Harvard Medical School has been published in EMBO Molecular Medicine.
Potential clinical impact
Because the inner ear’s hearing and balance systems share vulnerable cellular structures, a therapy that stabilises hair cells could prevent or delay both sensorineural hearing loss and vestibular disorders.
This represents a shift from compensatory devices such as hearing aids and cochlear implants toward a strategy that tackles the underlying cause.
Early intervention may be particularly important for children born with genetic conditions affecting hearing and balance. Preserving hair cell function could support typical speech, language and motor development and reduce reliance on long-term rehabilitation.
Expert view
“This work presents an exciting possibility in a growing body of research into stem cell and genetic therapies for hearing loss,” said Micaela Stonestreet, a Clinical Audiologist at Hearology®. “If similar results emerge in human trials, we could see a meaningful shift from managing symptoms to preventing progression.”
References
Breakthrough therapy treats genetic hearing and balance disorders - News story about the research
Could Gene Therapy Fix Hearing and Balance Problems? - Johns Hopkins Medicine discusses the potential of gene therapy in treating hearing and balance issues
NIH scientists improve hearing and restore balance in mice with inherited form of deafness - Previous report on gene therapy’s effect on mice from the National Institute on Deafness and Other Communication Disorders
The Future of Gene Therapy for Hearing Loss - The National Institute on Deafness and Other Communication Disorders (NIDCD) provides information on gene therapy for hearing loss
