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Results KAT6A mutations cause a decrease in histone H3 lysine 23 (H3K23) acetylation in human cells and mice To determine the effects of mutation of the KAT6A gene on histone acetylation, we generated human HEK293T cells carrying six different KAT6A variants observed in individuals with ARTHS using CRISPR/Cas9 and homology-directed repair (Tables S1, S2, and S3)

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The drug group did not change at all after 12 months and when retested 24 months after the start of the trial