Date: Friday, October 7, 2016
Time: 12:00 pm-1:00 pm
Speaker: Xin Tang, Ph.D.
Affiliation: Simons Postdoctoral Fellow, Rudolf Jaenisch Lab, Whitehead Institute and Picower Institute for Learning & Memory, MIT

Talk Title: Identification of Small Molecule Compounds to Treat Rett Syndrome with a Human Neuron-based Drug Screening Platform 
Abstract: A disruption in the balance between excitatory and inhibitory neuronal signaling (E/I balance) is observed in various neurological diseases including Rett Syndrome (RTT), a severe neurodevelopmental disorder with autism phenotypes. Our previous work reported a reduction in the level of KCC2, a neuron-specific gene crucial for the maintenance of E/I balance, in cultured human RTT patient-derived neurons and in brains of RTT mouse model. In the current project, we aim to identify drugs that increase KCC2 expression in human RTT neurons. We have applied CRISPR/Cas9 gene editing technologies to human embryonic stem cells to develop a chemical compound screening platform, and have identified and validated the first batch of KCC2 expression enhancer compounds from a screen of multiple compound libraries. We propose that results from our research will help the development of novel therapies that target KCC2 to treat RTT and other brain disorders with impairments in E/I balance.