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CRISPR-Based Synthetic Gene Circuits as Next Generation Gene Therapy of Inner Ear
CRISPR-Based Synthetic Gene Circuits as Next Generation Gene Therapy of Inner Ear
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机译:基于CRISPR的合成基因电路作为内耳的下一代基因疗法
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摘要
Aspects of the disclosure relate to synthetic regulatory systems comprising a multifunctional Cas nuclease and at least two guide RNAs (gRNAs) including a truncated gRNA and an multilayered regulatory control element. The synthetic regulatory system modulates endogenous gene expression, including transcriptional repression and transcriptional activation of one or more endogenous genes of a mammalian inner ear cell with multiple safety switches. Also provided herein are methods for modulating hearing sensitivity in damaged cells of the organ of corti.
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