Recombinant GLUT1 adeno-associated viral vector constructs and related methods for restoring GLUT1 expression

摘要

The present invention relates to recombinant Glut1 adeno-associated viral vector (rAAV) constructs and related methods for restoring Glut1 expression in Glut1 deficient mammals. In certain embodiments, the rAAV further comprises a chicken beta -actin promoter, wherein the rAAV can cross the blood brain barrier (BBB). In certain embodiments, the invention relates to compositions comprising any of the recombinant AAVs described herein. In certain embodiments, the present invention relates to a kit comprising a container housing comprising the composition described herein. In certain embodiments, the present invention relates to a method of restoring Glut1 transport in a subject''s BBB, comprising administering to the subject an effective amount of any of the recombinant AAV vectors described herein. In certain embodiments, the invention relates to a method of treating a Glut1 deficiency syndrome in a subject in need of such treatment.