A group at the University of California, Riverside have found a new potential approach for reducing symptoms of Fragile X syndrome (FXS) upon experimenting with young transgenic mice.
According to the findings in Neurobiology of Disease, FXS symptoms was reduced substantially upon inserting Fmr1 within the brains of rodents pre-fixed to lack the gene.
Upon examining the brain activity of the rodents for indications of stress or anxiety after exposed to certain stimuli, reactivation of the Fmr1 gene resulted in a cessation of FXS symptoms.
From the study: “These results indicate that postnatal deletion or re-expression of Fragile X mental retardation protein (FMRP) in excitatory neurons is sufficient to elicit or ameliorate structural and functional cortical deficits, and abnormal behaviors in mice, informing future studies about appropriate treatment windows and providing fundamental insights into the cellular mechanisms of cortical circuit dysfunction in FXS.”
“Our understanding of the time period during which these deficits manifest may allow for the development of therapeutic approaches that can potentially be targeted to impact specific cell types, circuits, and symptoms,” the authors also stated in the findings.
Funding for the research include the National Institute of Child Health and Human Development, the National Institute of Mental Health, and the Stroke National Research Service Award Fellowship.
