A Novel Gene Therapy Approach for Fragile X Syndrome

Time: 5:00 pm
day: Day One


  • The Diacylglycerol kinase kappa (DGKk) hypothesis model of Fragile X syndrome
  • Delivery of DGKk to the brain corrects disease phenotypes in FMR1 knockout mice
  • Development of AAV-DGKk as a gene therapy treatment for Fragile X syndrome