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  "authorName": "Taysha Gene Therapies",
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  "notes": "Includes a biological diagram of the synapse and a therapeutic approach summary (AAV9 + hSLC6A1 + Intrathecal delivery).",
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      "text": "The SLC6A1 gene encodes the GABA transporter 1 (GAT-1)\nPatients with SLC6A1 have a deficiency of GAT-1, resulting in decreased GABA transport and aberrant neuronal activity\nThe symptoms of SLC6A1 typically appear at 3-4 years and progressively worsen with age\nSLC6A1 has an estimated prevalence of 16,000 patients, which we believe is underestimation as there is an emerging epidemiology",
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