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  "authorName": "Taysha Gene Therapies",
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  "notes": "Includes a scientific diagram of glycogen branching and a bar chart showing Gys1 deficiency impact.",
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      "text": "AAV9 + GYS-miRNA + Intrathecal delivery",
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      "text": "In Lafora disease and APBD, glycogen chain elongation outpaces glycogen branching. Elongated glycogen molecules accumulate into toxic polyglucosan bodies. Downregulation of glycogen synthase (GYS) can prevent polyglucosan body formation. Taysha is pursuing a GYS-miRNA therapy for the treatment of Lafora and APBD. The estimated prevalence of GSDs is 20,000 patients in the US and EU.",
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