Gene therapy delivery compositions and methods for treating hearing loss

The present disclosure provides constructs comprising a coding sequence operably linked to a promoter that expresses a polynucleotide in an outer hair cell, where the coding sequence encodes a polypeptide (e.g., a heterologous polypeptide). Exemplary constructs include AAV constructs. Methods of tre...

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Bibliographische Detailangaben
Hauptverfasser: JIANG HAO, GRIBBLE KERRY D, LENTZ, DAVID, R, WU RAEANN
Format: Patent
Sprache:chi ; eng
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Zusammenfassung:The present disclosure provides constructs comprising a coding sequence operably linked to a promoter that expresses a polynucleotide in an outer hair cell, where the coding sequence encodes a polypeptide (e.g., a heterologous polypeptide). Exemplary constructs include AAV constructs. Methods of treating hearing loss and/or deafness using the disclosed constructs are also provided. 本公开提供了包含编码序列的构建体,所述编码序列可操作地连接至在外毛细胞中表达多核苷酸的启动子,其中所述编码序列编码多肽(例如,异源多肽)。示例性构建体包括AAV构建体。还提供了使用所公开的构建体来治疗听力损失和/或耳聋的方法。