University of Massachusetts;The Trustees of Columbia University in the City of New York
发明人:
DE VIVO, Darryl,MONANI, Umrao,GAO, Guangping,ENGELSTAD, Kristin
申请号：
EP16762530
公开号：
EP3268024A4
申请日:
2016.03.10
申请国别(地区):
EP
年份:
2018
代理人:
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 β-actin promoter wherein the rAAV is capable of crossing the blood-brain barrier (BBB). In certain embodiments, the present invention relates to a composition comprising any of the recombinant AAV's 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 methods of restoring Glut1 transport in the BBB of a subject, comprising administering to the subject an effective amount of any of the recombinant AAV vectors described herein. In certain embodiments, the present invention relates to a method of treating Glut1 deficiency syndrome in a subject in need thereof.
