Restoring Fragile X function with gene editing

 

Fragile X syndrome is the most prevalent genetic form of intellectual disability. There is no cure or treatment due in part to the complexity in the Fragile X syndrome neuronal circuitry. In this webinar, Dr. John Graef, Principal Scientist at Fulcrum Therapeutics, demonstrates how using CRISPR gene-editing and patient-derived cells Fulcrum can create the Fragile X syndrome phenotype in a dish. Moreover, this approach has enabled an estimate of the level of FMRP protein expression required to correct the observed Fragile X syndrome phenotype.