Neural deficits in PACS1 syndrome caused by dysregulated HDAC6 are corrected with targeted therapy

PACS1 syndrome is caused by an Arg203Trp mutation in PACS1. We show PACS1R203W dysregulates HDAC6 to disturb neuronal structure and function in vivo. Silencing PACS1R203W/HDAC6 reverses these deficits, suggesting this disorder is treatable.