With the use of the ASOs to target the SCN1A NAT, one study was effectively able to rescue the sense SCN1A gene expression, ameliorate neuronal excitability and seizures in a mouse model of DSan effect that almost entirely recapitulates the rescued phenotypes observed via CRISPRa treatment [74, 75]
Additionally, decreased expression of Sequestosome 1 (SQSTM1) in diabetic keratinocytes impairs autophagy-lysosome-mediated degradation of ACSL4, and increasing ACSL4 expression could drive ferroptosis [48]
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To broadly recapitulate the regulation of YBX1AMPK axis by -KG availability, we treated human cancer cells with cell-permeable DM-succinate and ITA