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  "documentTitle": "Taysha | IPO Presentation Deck | 66 slides",
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  "authorName": "Taysha Gene Therapies",
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  "notes": "Includes a visual mechanism of action diagram showing the silencing of Ube3a-ATS to restore Ube3a expression.",
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      "text": "Mechanism of action diagram showing maternal UBE3A loss and paternal UBE3A activation via shRNA",
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      "text": "Angelman syndrome is caused by the loss of function of the maternal copy of the UBE3A gene. Symptoms include severe cognitive dysfunction, absent speech, seizures, ataxia, gait disorder, microcephaly, and happy affect. Paternal copy is silenced.",
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      "text": "Taysha is developing gene therapies to activate the paternal UBE3A using an shRNA construct. Estimated prevalence is 30,000 patients in the US and EU.",
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      "text": "AAV9 + Anti-UBE3A-ATS shRNA + Intrathecal delivery",
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