In vivo xenotransplantation of patient iPSC-derived neurons in MECP2 neurodevelopmental disorders
Nona Merckx, Leïla Boubakar, Ryohei Iwata, Emir Erkol, Pierre Vanderhaeghen, Hilde Van Esch · FENS Forum 2024
Intellectual disability and autism spectrum disorders resulting from MECP2 gene alterations, such as Rett syndrome (RTT) and MECP2-duplication syndrome (MDS), present challenges in understanding the complex links between genetic alterations and cognitive deficits. Previous animal-based studies, though insightful, face translational barriers due to evolutionary differences in brain development between species, hindering direct application to humans.To address this, we established an innovative in vivo human neuronal model for RTT and MDS, utilizing patient-derived induced pluripotent stem cells
ePoster · Neuroscience