Gene Therapy for Glut1 Deficiency Syndrome: Upregulation of Cerebral Glucose at the Blood-Brain Barrier

Time: 1:30 pm
day: Day One


  • Glucose transporter 1 Deficiency Syndrome (Glut1 DS) is a
    neurodevelopmental genetic disorder caused by haploinsufficiency of
    SLC2A1 gene encoding Glut1
  • There is currently a high unmet need for a long-term therapy that does
    not bypass the utilization of glucose as an energy source in the brain
  • Demonstrating that rAAV9 mediated delivery of Glut1 in neonatal mice
    restores the function of the deficient Glut1 in the Glut1 DS mouse model