Transient peripheral blood transcriptomic response to ketamine treatment in children with ADNP syndrome.

ABSTRACT: Activity-dependent neuroprotective protein (ADNP) syndrome is a rare neurodevelopmental disorder resulting in intellectual disability, developmental delay and autism spectrum disorder (ASD) and is due to mutations in the ADNP gene. Ketamine treatment has emerged as a promising therapeutic option for ADNP syndrome, showing safety and apparent behavioral improvements in a first open label study. However, the molecular perturbations induced by ketamine remain poorly understood. Here, we investigated the longitudinal effect of ketamine on the blood transcriptome of 10 individuals with ADNP syndrome. Transcriptomic profiling was performed before and at multiple time points after a single low-dose intravenous ketamine infusion (0.5 mg/kg). We show that ketamine triggers immediate and profound ge

SUBMITTER: Buxbaum Grice AS 

PROVIDER: S-EPMC11272924 | biostudies-literature | 2024 Jul

REPOSITORIES: biostudies-literature