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Gene Therapy Restores Brain Function and Behavior in Fragile X Syndrome

An AAV-based therapy carrying a normal human version of the FMR1 gene to produce the Fragile X messenger ribonucleoprotein (FMRP) found that early treatment nor...

An AAV-based therapy carrying a normal human version of the FMR1 gene to produce the Fragile X messenger ribonucleoprotein (FMRP) found that early treatment normalized several measures of brain activity while improving social behavior, exploratory behavior, and cognitive flexibility. The post Gene Therapy Restores Brain Function and Behavior in Fragile X Syndrome appeared first on Inside Precision Medicine .