Gene repair technique can improve brain dysfunction in adults

Researchers have claimed that brain dysfunction such as seizure and memory impairments can be corrected after restoring certain protein level through gene repair techniques in adult patients.

A new study published in the Journal of eLife challenges the presumption that people born with developmental brain disorders such as severe autism will benefit from medical interventions only if treated during early childhood.

This may include intellectual disability, autism-like behaviors, disordered sensory processing, and epileptic seizures that don't respond to medication.

According to treatment that was conducted on adult mice, multiple improvements were seen. It suggests that having one broken copy of the gene not only harms the brain as it develops but also has effects in the adult brain.

"Our findings in mice suggest that neurodevelopmental disorders' disease course can be altered in adult patients. We can correct brain dysfunction related to seizure as well as memory impairments after restoring SynGAP protein levels in the adult animals," said Rumbaugh, a member of the research team.

Significantly, the paper offers a path to measure the effectiveness of potential medications or other therapies for neurodevelopmental disorders going forward.

Establishment of biomarkers that predict generalised improvements in brain function will be a critical step in advancing treatments for people with severe neurodevelopmental disorders, Rumbaugh said.

"The need for a treatment option is clear. Seizures typically become more frequent as children with SYNGAP1 gene disorders mature, and for many patients, those seizures do not respond to anti-epilepsy drugs," the researcher mentioned.

Rumbaugh continued, "Getting to know families affected by this severe disorder has been invaluable, and drives us to develop treatments that will improve the lives of both children and adults. It is encouraging that gene therapy techniques that increase pathologically low protein levels for other types of brain disorders are showing promise in the clinic now.