Taysha IPO Presentation Deck slide image

Taysha IPO Presentation Deck

Rett syndrome (RTT) is an X-linked neurodevelopmental disorder Characterized by mutations in methyl CpG-binding protein 2 (MeCP2), a protein that is essential for neuronal and synaptic function in the brain. Female heterozygous RTT patients are mosaic carriers of normal and mutated MECP2 RTT falls along a spectrum of MeCP2 activity and toxicity from gene therapies is linked to unregulated expression of MECP2 MECP2 expression must be regulated to correct the deficiency, while avoiding toxicity associated with overexpression T Skewed inactivation loss WT mosaicism WT WT WT WT normal (WT) WT WT WT WT WT WT Skewed inactivation overexpression 00 29
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