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The therapeutic interventions in ASD aimed at rescuing haploinsufficiency of individual genes could be developed to target all three levels of the central dogma of molecular biology: DNA, mRNA, and protein ( Fig

This elevation in NAD+ levels activates critical metabolic pathways including SIRT1 (sirtuin 1), a longevity-associated protein that regulates insulin sensitivity, mitochondrial function, and cellular stress resistance

For CRISPRa to work, single-guide RNAs (sgRNA) are designed to target regions between 1 to 1000 bp upstream of the transcription start site (TSS) within the promoter region of the target gene