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  "authorName": "Taysha Gene Therapies",
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  "notes": "The chart is a box-and-whisker plot comparing age of onset, death, and last report across multiple genetic conditions, highlighting SURF1.",
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      "text": "Box-and-whisker plot showing age of onset, death, and last report for various genetic conditions including SURF1.",
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      "text": "AAV9 + hSURF1 + Intrathecal delivery",
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      "text": "Deficiency in the SURF1 gene results in mitochondrial dysfunction and dysregulation of oxidative phosphorylation. SURF1 mutations are implicated in Leigh syndrome as well as Charcot-Marie Tooth 4K. Symptoms appear in late infancy and include episodic neurological regression, movement disorder, and respiratory failure. The estimated prevalence of SURF1 is 300 patients in the US and EU.",
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