Researchers develop breakthrough gene therapy to restore hearing and balance
Research led by PhD student Roni Hahn and Prof. Karen Avraham, in collaboration with researchers from Boston Children’s Hospital and Harvard Medical School
A team led by Prof. Karen Avraham and PhD student Roni Hahn has successfully restored hearing and balance in a mouse model of genetic deafness. The study, conducted in collaboration with Harvard Medical School and Boston Children's Hospital, was published in EMBO Molecular Medicine and featured on its cover.
The team addressed defects in the CLIC5 gene, which encodes a protein crucial for stabilizing the structure and organization of hair cell stereocilia - the inner ear's sensory receptors. Mutations in CLIC5 lead to progressive hair cell degeneration, causing autosomal recessive hearing loss and severe balance dysfunction.
To reverse these effects, the researchers used engineered viral vectors to deliver a healthy copy of the Click5 gene directly into the inner ears of newborn mice with CLIC5 deficiency. The treatment restored the normal structure of sensory hair cells, prevented further degeneration, and significantly improved both hearing and balance. Treated mice responded to sounds and showed marked improvements in motor coordination and movement behavior.
The findings provide strong evidence that gene therapy could one day offer an effective treatment for people with CLIC5-related deafness and balance disorders, while also expanding the growing toolbox of gene-based therapies for inherited hearing loss.
Read the full paper:
AAV gene therapy rescues hearing and balance in a model of CLIC5 deafness
Published in: EMBO Molecular Medicine
Authors: Roni Hahn, Shahar Taiber, Olga Shubina-Oleinik, Gwenaëlle S G Géléoc, Jeffrey R Holt & Karen B Avraham
https://link.springer.com/article/10.1038/s44321-025-00275-7
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