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Gene therapy has restored hearing for up to 18 months in mice that were born deaf

Gene therapy has restored hearing for up to 18 months in mice that were born deaf

 Prof. Lawrence Lustig
Prof. Lawrence Lustig at the University School of Medicine San Francisco and associates have reported that the newly born mice that lack the gene encoding the transporter VGLUT3 are deaf because the absence of this gene renders inner hair cells of the ear’s cochlea incapable of sending electrical signals to the brain. This exciting piece of news published online in the August 1 issue of Nature (488, 9, 02 August 2012) under the title “Gene fix repairs hearing’ summarized the main work of the team published in the journal “Neuron” (75:2, 283-293, 26th July, 2012). The authors have demonstrated that through replacing this defective gene it’s possible to restore the hearing ability for up to 18 months in mice that were born deaf. To date Efforts to restore hearing with gene therapy have been met with limited success. The team has used an specific Adeno-associated virus type 1 (AAV1) delivery system to deliver the Vglut3 gene into the cochleas. As VGLUT3 is also associated with a rare form of deafness in human, this can be a significant advance toward gene therapy of human deafness.

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