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  "authorName": "Taysha Gene Therapies",
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      "text": "AAV9/MECP2 caused dose-dependent side effects after intraCSF administration in WT and KO mice. We have developed a novel miRNA-responsive target sequence (miRARE) that regulates the expression of the MECP2 transgene. Our approach provides a superior therapeutic profile to that of competitor unregulated MECP2 gene replacement.",
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