Scientists in the US have shown that correcting the protein deficiency caused by a genetic form of autism spectrum disorder (ASD) in adult mice can improve some of the characteristic symptoms, even though the treatment is carried out well past what has traditionally been thought of as the critical window of early brain development. The studies, carried out in a mouse model of human ASD caused by defects in the SYNGAP1 gene, found that restoring normal SynGAP protein levels in adult animals improved behavioral and electrophysiological measures of both memory and seizure. The authors suggest that future gene therapies for genetic causes of neurodevelopmental disorders (NDDs) such as ASD, intellectual disability (ID), and epilepsy, may also be effective in adult patients.
“Our findings in mice suggest that neurodevelopmental disorders’ disease course can be altered in adult patients,” said research lead Gavin Rumbaugh, PhD, an associate professor in the department of neuroscience at Scripps Research in Florida. “We can correct brain dysfunction related to seizure as well as memory impairments after restoring SynGAP protein levels in the adult animals.”
Rumbaugh’s team, together with colleagues at the University of Texas at Austin, and the Jan and Dan Duncan Neurological Research Institute and department of pediatrics at Baylor College of Medicine, report their results in eLife, in a paper titled, “Re-expression of SynGAP Protein in Adulthood Improves Translatable Measures of Brain Function and Behavior.”