Positive effects of low dose IMPX977 on Rett syndrome related MeCP2 targeted-genes

ABSTRACT Objective To investigate the effect of IMPX977 on MeCP2 targeted-genes and the feasibility of IMPX977 acting as a therapeutic candidate drug for Rett syndrome by genomewide transcription profiling. Methods Rats' cortex of control group, IMPX977-treated low-dose group (10 mg/kg), and IMPX977-treated high-dose group (30 mg/kg) were collected and RNA was extracted from the tissues. Then, RNA was subjected to RNA-sequencing. Gene ontology (GO) and kyoto encyclopedia of genes and genomes (KEGG) were used in functional enrichment analysis of differentially expressed genes. Results Six MeCP2 targeted-genes were identified in the low/control categories, but not in the high/control categories. Conclusion Low-dose treatment of IMPX977 (10 mg/kg) showed a positive effect on MeCP2 targeted-genes and it may serve as a drug candidate for Rett syndrome therapy with proper dosage.