Penn State 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. The research, led by Gong Chen, professor of biology and the Verne M. Willaman Chair in Life Sciences at Penn State, could lead to a new treatment for Rett Syndrome and other forms of autism-spectrum disorders. A paper describing the research was published on Jan. 4, 2016, in the online Early Edition of the journal Proceedings of the National Academy of Sciences.
“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 Chen. “Therefore, the new drug target discovered in this study might have direct clinical implication in the treatment of Rett Syndrome and potentially for other autism-spectrum disorders as well.”