"Breakthroughs in Treating Autism and Aging with Innovative Approaches"
Researchers have discovered a potential new treatment for Fragile X Syndrome (FXS), a leading cause of autism spectrum disorders, by contracting the CGG trinucleotide repeat in the FMR1 gene, thereby restoring the essential FMRP protein expression necessary for brain development. The process required the presence of inhibitors of two kinases, MEK and BRAF, to induce repeat contraction and full FMR1 reactivation, effectively reducing the expanded CGG repeats and stimulating the body's own DNA repair mechanisms. The results mark a significant stride towards a potential "one-and-done" treatment for FXS.