| Abstrakt: |
A recent study conducted by researchers at the University of Massachusetts Chan Medical School has identified a potential therapeutic approach for Fragile X Syndrome (FXS), a neurological disorder caused by the epigenetic silencing of the FMR1 gene. The study found that inhibiting the histone methyltransferase EZH2, using small molecules or antisense oligonucleotides (ASOs), can reactivate FMR1 expression and correct molecular and electrophysiological abnormalities in FXS neurons. While EZH2 inhibitors have limited therapeutic use due to their inability to efficiently cross the blood-brain barrier, the development of ASO-based approaches shows promise as a treatment option for FXS. [Extracted from the article] |
