Studies in animal models and postmortem human tissues have provided key insights into the pathogenesis of AD1,2,9. However, mouse models of AD do not recapitulate many AD features, and postmortem human brain tissues have characteristics of end-stage disease that may not be present at earlier stages1,2,9. Until recently, studies of the cellular and molecular mechanisms of AD have been hindered by the lack of access to live human neurons. Now, induced pluripotent stem cells (iPSCs) derived from human somatic cells with AD-linked mutations or polymorphisms, together with gene-editing techniques, are promising in vitro models for studying disease pathogenesis in relevant cell types, including human neurons10–15.
