Using hiPSCs to model neuropsychiatric copy number variations (CNVs) has potential to reveal underlying disease mechanisms

摘要: Schizophrenia is a neuropsychological disorder with strong heritable component; genetic risk for schizophrenia conferred by both common variants of relatively small effect and rare high penetrance. Genetically engineered mouse models can recapitulate variants, displaying some behavioral defects associated schizophrenia; however, these cannot the full architecture underlying disorder. Patient-derived human induced pluripotent stem cells (hiPSCs) present an alternative approach studying in context all other alleles. Genome editing technologies, such as CRISPR-Cas9, enable generation isogenic hiPSC lines which to examine functional contribution single within any background. Studies using hiPSCs have potential identify commonly disrupted pathways allow identification new therapeutic targets. This article part Special Issue entitled SI:StemsCellsinPsychiatry.