Gene Therapy for Glut1 Deficiency Syndrome: Upregulation of Cerebral Glucose at the Blood-Brain Barrier
Time: 1:30 pm
day: Day One
Details:
- 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
