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GENE THERAPY CONSTRUCTS AND METHODS FOR TREATMENT OF HEARING LOSS
GENE THERAPY CONSTRUCTS AND METHODS FOR TREATMENT OF HEARING LOSS
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机译:基因治疗的结构和方法治疗听力损失
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摘要
Disclosed are compositions and methods that useful in the treatment and/or prevention of hearing loss 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.
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