Compositions and methods for treating sensory neurological hearing loss using orthodontic protein dual carrier system

The present disclosure provides compositions and methods for the treatment of sensory neurological hearing loss and auditory neuropathy, in particular disease forms associated with ear-dontic protein (OTOE) mutations, by means of OTOE gene therapy. The present disclosure provides various compositions comprising a first nucleic acid vector comprising a polynucleotide encoding an N-terminal portion of an OTOE isoform 5 protein and a second nucleic acid vector comprising a polynucleotide encoding a C-terminal portion of an OTOE isoform 5 protein. These vectors are useful for increasing the expression of OTOE in a subject, such as a human subject suffering from sensory neurological hearing loss. 本公开提供了借助于OTOE基因疗法治疗感觉神经性听力损失和听神经病变、特别是与耳畸蛋白(OTOE)突变相关的疾病形式的组合物和方法。本公开提供了多种组合物，所述组合物包括含有编码OTOE同功型5蛋白的N末端部分的多核苷酸的第一核酸载体和含有编码OTOE同功型5蛋白的C末端部分的多核苷酸的第二核酸载体。这些载体可用于增加受试者、例如患有感觉神经性听力损失的人类受试者中OTOE的表达。