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Gene Therapy for Hearing Loss Reversal

Hearing loss has been linked to mutations in at least 100 different genes, but up to 16% of genetic hearing loss can be traced to just one gene, STRC, the second most common genetic cause. A first-of-its-kind gene therapy technique developed at Boston Children’s Hospital successfully replaced the mutated protein, stereocilin, in the inner ear and reversed severe hearing loss in mice — sometimes to normal levels of hearing.

Gene Therapy Used to Restore Hearing and Balance in Mice with Usher Syndrome

In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of Medicine showed that gene therapy was able to restore balance and hearing in genetically modified mice that mimic Usher syndrome.

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Partial Hearing and Balance Restored in Deaf Mice Using New Gene-delivery Therapy

Using a novel form of gene therapy, scientists from Harvard Medical School and the Massachusetts General Hospital have managed to restore partial hearing and balance in mice born with a genetic condition that affects both. The new model reportedly overcomes a long-standing barrier to accessing hair cells which have been notoriously difficult to treat with previous gene-delivery techniques.

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