The dawn of a kind of autism gene carrier-early gene therapy

Fragile X syndrome (FXS) is one of the main genetic causes of autism.

Researchers at the University of California, Riverside published a report in the journal Neurobiology of Disease that they implanted Fmr1 into a very young genetically modified gene without the FXS gene.

"Our work shows the beneficial effects of reactivating the Fmr1 gene, which will be very welcome news for young children living in an FXS environment," said Iryna M.

In their research, Ethell’s laboratory worked with psychology professor Khaleel A.

"For humans, the first 3-5 years is a critical period for brain development," Ethell said

Like the human brain, the mouse brain also has excitatory neurons and inhibitory neurons

Ethell and two colleagues recently published a review article in the journal Nature Neuroscience , stating that inhibitory neuronal dysfunction is a common pathology in genetic diseases associated with autism spectrum disorder (ASD)

"In the current study, we inserted the Fmr1 gene into the excitatory neurons in the second and third weeks after birth in mice," Ethell said

The way that Ethell and her team introduced the Fmr1 gene into the brain of mice is different from the way that the gene is potentially introduced into the brain of humans

She said: “FXS is usually diagnosed in the early stages of humans

Next, the research team will focus on restoring the function of the adult FXS brain

Ethell said: "The main challenge is that the adult brain is not that plastic

Neurobiology of Disease

DOI

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