Hard cilia protein promoter and application thereof
The present disclosure provides STRC promoters useful for expressing a desired expression product in hair cells that endogenously express hard ciliin (STRC), including cochlear hair cells and vestibular hair cells, as well as vectors containing the promoters. The STRC promoters described herein can...
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Format: | Patent |
Sprache: | chi ; eng |
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Zusammenfassung: | The present disclosure provides STRC promoters useful for expressing a desired expression product in hair cells that endogenously express hard ciliin (STRC), including cochlear hair cells and vestibular hair cells, as well as vectors containing the promoters. The STRC promoters described herein can be operably linked to polynucleotides encoding heterologous expression products, such as transgenes, and are used to treat subjects suffering from or at risk of developing hearing loss or vestibular dysfunction.
本公开提供了可用于在包括耳蜗毛细胞和前庭毛细胞在内的内源性表达硬纤毛蛋白(STRC)的毛细胞中表达所需表达产物的STRC启动子,以及含有所述启动子的载体。本文所述的STRC启动子能够被可操作地连接至编码异源表达产物的多核苷酸,诸如转基因,并用于治疗患有听力损失或前庭功能障碍或处于发展成听力损失或前庭功能障碍的风险中的受试者。 |
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