Topic: patient-derived induced pluripotent stem cells

We are investigating the developmental origins of alterations in striatal interneurons observed in brain of patients diagnosed with Tourette’s Syndrome using organoid models cultured from patient-derived induced pluripotent stem cells. Here we provide a first characterization of organoids differentiated using a new protocol aimed to induce ventral forebrain fates in 7 iPSC lines from neurotypical adults. During neural induction with dual SMAD inhibition, patterning was carried out over 5 days using the Wnt-antagonist XAV939 alone in our standard dorsal forebrain protocol, and in combination wi

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