发明名称 |
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 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. |
申请公布号 |
WO2016145217(A1) |
申请公布日期 |
2016.09.15 |
申请号 |
WO2016US21810 |
申请日期 |
2016.03.10 |
申请人 |
THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK;UNIVERSITY OF MASSACHUSETTS MEDICAL CENTER |
发明人 |
DE VIVO, Darryl;MONANI, Umrao;GAO, Guangping;ENGELSTAD, Kristin |
分类号 |
A61K38/16;A61K48/00;A61P25/00;A61P25/28 |
主分类号 |
A61K38/16 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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