In vitro Rett syndrome model based on MeCP2 knockdown in iPSC-derived neurons
Rie Yamoto, Mika Nakao, Toru Hazama, Toshihiko Hosoya · FENS Forum 2024
Rett Syndrome (RTT) is a X-linked neurodevelopmental disorder that predominantly affects girls. The detailed disease mechanisms remain unclear and fundamental treatments are yet to be established. The majority of typical RTT cases is triggered by mutations in the methyl CpG-binding protein 2 (MeCP2) gene. For the analyze of the disease mechanisms and drug development, in vitro models based on iPSC-derived cells with diseased MeCP2 are being investigated. In addition to MeCP2 mutations RTT is also affected by the genetic background, and thus it is necessary to analyze the effect of MeCP2 modifi
ePoster · Neuroscience