PROCEDES ET COMPOSITIONS POUR L'ADMINISTRATION DE VECTEURS VIRAUX A TRAVERS LA BARRIERE HEMATO-ENCEPHALIQUE

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专利权人:

THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL

发明人:

LI, CHENGWEN,SAMULSKI, RICHARD JUDE

申请号：

CA3085945

公开号：

CA3085945A1

申请日:

2018.12.19

申请国别(地区):

CA

年份:

2019

代理人:

摘要：

Disclosed herein is an adeno-associated virus (AAV) particle comprising a surface-bound peptide that enhances transduction of cells across the blood-brain barrier (BBB). Also disclosed herein is a modified AAV capsid protein comprising an insertion of a polypeptide that enhances transduction of cells across the BBB, and an AAV particle comprising the modified AAV capsid protein. Specific peptides are provided. Pharmaceutical formulations and method of administering/delivering a nucleic acid to a cell of the brain and/or central nervous system are also disclosed.

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