Therapeutics: Protein tyrosine phosphatase 1B (PTP-1B; PTPN1); methyl CpG binding protein 2 (MECP2; RTT)

Mouse and patient sample studies suggest inhibiting PTP-1B could help treat Rett syndrome. PTP-1B levels were higher in fibroblasts from Rett syndrome patients than in cells from healthy subjects, and forebrain levels of PTP-1B transcripts and protein were higher in male and female mutant MECP2 mouse models of Rett syndrome than in wild-type mice. In the mouse models, a PTP-1B inhibitor increased glucose tolerance, extended survival of male mice and decreased behavioral deficits in female mice compared with vehicle. Next steps could include testing PTP-1B inhibition in additional models of Rett syndrome...