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In a groundbreaking advancement in the treatment of fragile X syndrome (FXS), researchers have successfully deployed gene therapy targeting the FMR1 gene, restoring key neurologica...
A gene therapy aimed at correcting fragile X syndrome by restoring the missing FMR1 protein has demonstrated multiple disease-relevant improvements in a mouse model, according to a...
The therapy restored FMRP expression in crucial brain regions, resulting in reduced audiogenic seizure susceptibility, normalized sensory hyperactivity, and complete reversal of el...
Can an overactive synaptic protein hold the key to treating the most common genetic cause of autism? A new study identifies the protein EPAC2 as a potent drug target for reversing...
Researchers identified the synaptic protein EPAC2 as a potential therapeutic target for fragile X syndrome, and showed that blocking EPAC2 in an FXS mouse model restored abnormal p...
A new study delivered the first empirical proof that severe genetic mutations can completely eliminate the natural biological protection females hold against autism spectrum disord...
A new study demonstrated a cellular neighborhood effect, where mutant cells actively warp the gene expression of neighboring healthy neurons, revealing that early circuit wide-dama...
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