A novel drug target has been discovered by the Penn State scientists to rescue functional deficits in human nerve cells derived from patients with Rett Syndrome, a severe form of autism-spectrum disorder.
According to lead researcher Gong Chen, the most exciting part of this research is that it directly uses human neurons which originated from Rett Syndrome patients as a clinically-relevant disease model to investigate the underlying mechanism.
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.
The study has been published in the online Early Edition of the journal Proceedings of the National Academy of Sciences.
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