遗传性听力损失的基因治疗

Devin S. McDougald, L. Cunningham, W. Chien
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引用次数: 0

摘要

目前已确定70多个基因的突变可导致人类遗传性听力损失。关于听力损失的遗传学知识的不断扩大,为基因疗法的发展打开了大门,旨在通过向由这些突变引起的听力损失患者的内耳提供校正DNA来恢复听力功能。这篇综述总结了一些最近的进展,目标是发展基因治疗作为治疗遗传性听力损失的人类。
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Gene Therapy for Hereditary Hearing Loss
Mutations in over 70 genes have now been identified as causing hereditary hearing loss in humans. This expanding knowledge about the genetics of hearing loss opens the door for the development of gene therapies aimed at restoring hearing function by delivering corrective DNA to the inner ears of patients with hearing loss caused by these mutations. This review summarizes some of the recent advances toward the goal of developing gene therapy as a treatment for hereditary hearing loss in humans.
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