GENE THERAPY SYSTEMS AND RELATED METHODS FOR TREATMENT OF HEARING LOSS
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United States of America Patent
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Jun 11, 2020
app pub date -
Feb 5, 2020
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Mar 17, 2017
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Abstract
The present disclosure describes gene therapy systems, and related methods, useful for treating and/or preventing deafness caused by genetic mutation of the TMPRSS3 gene or the LOXHD1 gene. The compositions and methods disclosed herein use adeno-associated viral (AAV) vector gene delivery of TRMPSS3 or LOXHD1 into the inner ear to restore activity of the TMPRSS3 gene or the LOXHD1 gene, respectively, promote hair cell survival and restore hearing in patients suffering from hearing loss. As disclosed herein, the systems and methods may utilize a combination of gene therapy (e.g., molecular therapeutics) for hearing loss caused by a genetic mutation together with implantation of a cochlear implant.
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