Neural deficits in PACS1 syndrome caused by dysregulated HDAC6 are corrected with targeted therapy
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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.
创建时间：
2023-10-19
