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Lab Reports
September 5, 2012

Gene Therapy for Hearing Loss

JAMA. 2012;308(9):853. doi:10.1001/2012.jama.10869

Through gene therapy, researchers have reversed inherited genetic hearing loss in mice, representing an important milestone in the quest to treat genetic deafness in humans (Akil O et al. Neuron. doi: 10.1016/j.neuron.2012.05.019 [published online July 25, 2012]).

Investigators at the University of California, San Francisco, and colleagues used mice with hereditary deafness caused by a mutation in the gene that encodes the vesicular glutamate transporter 3 (VGLUT3) protein, which is crucial for inner hair cells to send signals that enable hearing. Previous research indicates that a mutation in the human version of this gene may play a role in some cases of congenital hearing loss in people.