Researchers at UMass Chan Medical School and RUSH University Medical Center have developed an antisense therapy that can restore production of the protein FMRP in cell samples from patients with fragile X syndrome. Fragile X syndrome is the most common form of inherited intellectual disability and a frequent cause of autism. The researchers discovered that aberrant alternative splicing of messenger RNA (mRNA) plays a major role in the development of the syndrome. By targeting the mis-splicing of the mRNA, the researchers were able to restore normal levels of FMRP in the cells. They used antisense oligonucleotides (ASOs), short DNA sequences that bind to target mRNA and correct the splicing errors. The findings offer hope for the development of a therapy for fragile X syndrome. The researchers plan to secure a partnership with a commercial enterprise to advance the ASO work into human clinical trials. The study was funded by the National Institutes of Health, the Simons Foundation for Autism Research, the FRAXA Research Foundation, and the UMass Chan BRIDGE Fund.


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