GSE159859
Patient-derived brain organoids uncover reversible cellular abnormalities in Pitt-Hopkins Syndrome II
We evaluate cellular phenotypic abnormalities and alterations in molecular pathways due to mutations in the gene TCF4 in cells derived from Pitt-Hopkins patients, in comparison with respective controls (parents). Overall design: Comparison between RNA-Seq libraries of neural progenitor cells of low passage from Pitt-Hopkins Syndrome patients and respective parents of matching sex. Libraries for each individual include three biological replicates.