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Updated: Jun 24, 2025

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse
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Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse

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双边基因疗法在患有自体衰退性聋症的儿童 9:单臂试验结果

Hui Wang1,2, Yuxin Chen1,2, Jun Lv1,2,3,4

  • 1ENT Institute and Otorhinolaryngology Department of Eye & ENT Hospital, Fudan University, Shanghai, China.

Nature medicine
|June 5, 2024
PubMed
概括

使用腺相关病毒 (AAV) 的双耳基因疗法显示出治疗遗传性聋 (DFNB9) 的前景. 这项研究发现,儿科患者的双边听力恢复和语音感知改善,没有报告严重不良事件.

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Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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