RECOMBINANT GLUT1 ADENO-ASSOCIATED VIRAL VECTOR CONSTRUCTS AND RELATED METHODS FOR RESTORING GLUT1 EXPRESSION

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United States of America Patent

SERIAL NO

15556412

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Abstract

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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.

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Patent Owner(s)

Patent OwnerAddress
THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK412 LOW MEMORIAL LIBRARY 535 WEST 116TH STREET MAIL CODE 4308 NEW YORK NY 10027

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
DE, VIVO Darryl New York, US 2 1
ENGELSTAD, Kristin Bronx, US 2 1
GAO, Guangping Westborough, US 186 6157
MONANI, Umrao Oradell, US 5 1

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