In this composite image, a human nerve cell derived from a patient with Rett syndrome shows significantly decreased levels of KCC2 compared to a control cell.
Image: Gong Chen lab, Penn State University
Penn State University scientists have discovered a novel drug target and have rescued functional deficits in human nerve cells derived from patients with Rett Syndrome, a severe form of autism-spectrum disorder. It could lead to a new treatment for Rett Syndrome and other forms of autism-spectrum disorders, ASD.
“The most exciting part of this research is that it directly uses human neurons that originated from Rett Syndrome patients as a clinically-relevant disease model to investigate the underlying mechanism,” said Dr. Chen...
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