New gene-editing therapy could help treat brain disorders in unborn babies

In order to prevent brain disorders and disabilities in children, researchers have figured out to perform gene therapy on embryos, a technique that has never been applied before.

Researchers at the University of North Carolina have claimed to found a cure for brain disorders in children that can lead to memory loss, learning disabilities, seizures, inability to feel pain and lack of speech.

Angelman syndrome can be spotted in unborn babies, but cannot be prevented. In affected children, one of their maternal genes called UBE3A silences a crucial paternal gene leading to debilitating, life-long symptoms.

With the help of powerful gene-editing tool CRISPR-Cas9, researchers were successfully able to switch on the muted paternal gene in mice embryos, which were then born with no trace of the disorder, explained Daily Mail.

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The treatment involved doctors injecting the fetus’ brain with a harmless virus that infects the neurons and delivers a suite of molecules that correct the genetic faults.

The tests in mice showed that when the gene therapy was given in the womb, it activates UBE3A in key parts of the brain. Their therapy switched the gene on in the cortex, which is vital for cognition, the hippocampus that controls memory, and the cerebellum, which controls movement.

Also, The Guardian reported, the team tested the therapy on human brain cells grown in a petri dish and showed similar results. “It really does raise the possibility that this gene therapy might be usable in humans,” said researcher Mark Zylka.

Gene therapy technique to directly inject into the brains of fetuses has never been done before, thus the team believes this could be an important step to treat common disorders such as autism.

“For many of these neurodevelopmental disorders, where the brain has been developing inappropriately because of a gene mutation, the best time to intervene is probably going to be early, very early,” Zylka said.

“People are getting this diagnosis, and there’s nothing to be done, they have to make pretty difficult decisions at that point,” Zylka concluded. “This may give them another option.”