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A human cellular model of a prototypical form of intractable childhood epilepsy supports selective impairment of inhibitory neurons as a key pathophysiological mechanism.

An inducedpluripotent stem cell (iPSC)-based model of KCNQ2-associated developmental epileptic encephalopathy suggests that disease is driven by dyshomeostaic neuronal mechanisms that are downstream of loss of M-current.

Alterations in mTOR signaling drive similar dysregulations in the critical mid-fetal neurodevelopmental processes of neurite outgrowth and cell migration in two distinct subsets of autism, idiopathic and 16p11.2 deletion.

Human liver organoids provide a patient-derived platform to interrogate host and viral mechanisms of HBV replication, perform anti-HBV and toxicity drug screens, and investigate the molecular determinants of related tumorigenesis.

Proteomic analysis of brain organoids from creatine transporter deficiency (CTD) patients enhance the understanding of CTD offering potential therapeutic targets and a robust foundation for continued research in the field.

Stem-cell-based neural replacement, in combination with optogenetic stimulation, could represent a translationally viable therapeutic strategy to overcome atrophy and paralysis of targeted muscles in people living with amyotrophic lateral sclerosis.

iMyoblasts, a novel iPS-derived PAX3 muscle stem cell for gene editing, muscle engraftment, and stem cell therapeutics to treat muscle injury and muscular dystrophies.