Gene repair improves memory and seizures in adult autism model
"by The Scripps Research Institute"
"by The Scripps Research Institute"
Quote:"Our findings in mice suggest that neurodevelopmental disorders' disease course can be altered in adult patients," Rumbaugh says. "We can correct brain dysfunction related to seizure as well as memory impairments after restoring SynGAP protein levels in the adult animals."
Significantly, the paper offers a path to measure the effectiveness of potential medications or other therapies for neurodevelopmental disorders going forward. Electrographic spikes between seizures is an indicator of epilepsy. In their paper, the scientists looked at human EEG data collected from a SYNGAP1 disorder patient registry and found that the appearance of these spikes were much more likely to occur during sleep. Similar findings were observed from mouse models of SYNGAP1 disorder, offering a useful endpoint. Establishment of biomarkers that predict generalized improvements in brain function will be a critical step in advancing treatments for people with severe neurodevelopmental disorders, Rumbaugh says.
The need for a treatment option is clear, Rumbaugh says. Seizures typically become more frequent as children with SYNGAP1 disorders mature, and for many patients, those seizures do not respond to anti-epilepsy drugs.
'Historically, we may regard materialism as a system of dogma set up to combat orthodox dogma...Accordingly we find that, as ancient orthodoxies disintegrate, materialism more and more gives way to scepticism.'
- Bertrand Russell
- Bertrand Russell