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Conversely, overexpression of HNF4 in HNF4-negative SNU449 cells strongly induced the luciferase activities from reporters containing the consensus (WT) HNF4 binding sites of MAT1A , BHMT , or CBS promoters, but this induction was significantly reduced when the binding sites were mutated (Mut) (Supplementary Fig

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The trade-off is higher cost and the requirement for clinical administration

The primary outcome is to identify the maximum dose tolerated, and the second aim is to evaluate neurological outcome