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Motor neuron-specific expression vectors
Motor neuron-specific expression vectors
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机译:运动神经元特异性表达载体
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摘要
The present disclosure relates to nucleic acid promoter sequences that are able to specifically express genes operatively linked to the promoter in brainstem and spinal motor neuron cells, and to methods for using such promoters to selectively express genes in motor neurons in vitro and in vivo. It is based, at least in part, on the discovery that the nucleic acid of SEQ ID NO: 1 functioned as a motor neuron-specific promoter and was successful in expressing transgenes in motor neuron cells in vivo. The present disclosure also relates to compositions that can increase the activity or expression level of miR-218 and to compositions that can decrease the expression of miR-218 target nucleic acids.
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机译:本公开涉及能够特异性表达与脑干和脊髓运动神经元细胞中的启动子可操作连接的基因的核酸启动子序列,并且涉及使用此类启动子在体外和体内选择性地在运动神经元中表达基因的方法。其至少部分基于以下发现:SEQ ID NO:1的核酸充当运动神经元特异性启动子,并且在体内成功地在运动神经元细胞中表达转基因。本公开还涉及可以增加miR-218的活性或表达水平的组合物,并且涉及可以降低miR-218靶核酸的表达的组合物。
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