September 2025: Avraham: Breakthrough gene therapy offers hope for genetic hearing loss
Prof. Karen Avraham, Edmond J. Safra member, Roni Hahn, a PhD student in her lab, and colleagues from Boston Children’s Hospital and Harvard Medical School, have developed a promising gene therapy that could one day restore hearing and balance in individuals affected by genetic inner ear disorders.
Prof. Karen Avraham, Edmond J. Safra member (Medical & Health Sciences), Roni Hahn, a PhD student in her lab, and colleagues from Boston Children’s Hospital and Harvard Medical School, have developed a promising gene therapy that could one day restore hearing and balance in individuals affected by genetic inner ear disorders.
The therapy targets the CLIC5 gene, which plays a critical role in maintaining sensory hair cells in the inner ear. These cells are essential for detecting sound and maintaining balance, and their degeneration leads to hearing and balance impairments.
The researchers’ innovative approach uses a self-complementary viral vector to deliver a healthy copy of the gene directly into the affected cells, showing faster and more efficient results than previous methods.
The study was published in the journal EMBO Molecular Medicine and featured on its cover page. The findings mark a significant step toward developing gene therapies for inherited hearing disorders, and may also inform treatments for other sensory and neurological conditions.
The study received wide media coverage: JPOST, Israeleconomico, Hearingreview, Medicalxpress, Latestly,Tribuneindia, MIRAGE, Azertag, REPUBLIC, Worldisraelnews, Bignewsnetwork, Lokmattimes, Thearabianstories, Mechnicalmind.
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