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Penn State University researchers have made a groundbreaking discovery: a potential treatment for Autism.
The university – along with the publication of a research paper in the Proceedings of the National Academy of Sciences – announced Monday that Penn State scientists 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 was led by Gong Chen, a professor of biology and the Verne M. Willaman Chair in Life Sciences at PSU. The discovery could be the starting point for a new treatment of Rett Syndrome.
According Onward State, “Rett Syndrome is a rare neurological disorder that almost exclusively affects females, leaving sufferers with no verbal skills, seizures, scoliosis, growth failure, and other complications.”
As expected, the research was rather extensive,
Chen, along with his team, was able to study nerve cells of patients with Rett Syndrome, finding a mutation in the MECP2 gene and a specific and important molecule that these nerve cells were lacking. That molecule, KCC2, is vital in normal nerve cell function and brain development, according to the report. He believes that putting that molecule into Rett neurons could return nerve cell function to normal and treat the syndrome.
“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,” Chen said. “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 team also discovered that diseased nerve cells could be treated with insulin-like growth factor 1 — or IGF1, a protein hormone that plays a vital role in childhood growth. “The study showed that treating the nerve cells with this protein ‘elevated the level of KCC2 and correcting the function of the GABA neurotransmitter.’ GABA contributes to motor control, vision, and other important functions.”
We have no idea what any of this means, but it sounds promising. Scientists, you know what I mean?
[H/T: Onward State]
