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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Hauptverfasser: JIANG HAO, GRIBBLE KERRY D, LENTZ, DAVID, R, WU RAEANN
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creator JIANG HAO
GRIBBLE KERRY D
LENTZ, DAVID, R
WU RAEANN
description 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。
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
title Gene therapy delivery compositions and methods for treating hearing loss
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