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    COMPOSITIONS AND METHODS FOR ALTERING TISSUE SPECIFICITY AND IMPROVING AAV9-MEDIATED GENE TRANSFER
    6.
    发明申请
    COMPOSITIONS AND METHODS FOR ALTERING TISSUE SPECIFICITY AND IMPROVING AAV9-MEDIATED GENE TRANSFER 审中-公开
    用于改变组织特异性并改善AAV9介导的基因转移的组合物和方法

    公开(公告)号：WO2012112832A1

    公开(公告)日：2012-08-23

    申请号：PCT/US2012/025550

    申请日：2012-02-17

    申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA ,  WILSON, James, M. ,  BELL, Christie, L. ,  VANDENBERGHE, Luc, H.

    发明人： WILSON, James, M. ,  BELL, Christie, L. ,  VANDENBERGHE, Luc, H.

    IPC分类号： C12N15/861 ,  A61K48/00

    CPC分类号： A61K31/7088 ,  A61K31/685 ,  A61K38/47 ,  C12N15/86 ,  C12N2750/14043 ,  C12N2750/14045 ,  C12N2810/6027

    摘要： A method of altering the targeting and/or cellular uptake efficiency of an adeno- associated virus (AAV) viral vector having a capsid containing an AAV9 cell surface binding domain is described. The method involves modifying a clade F cell surface receptor which comprises a glycan having a terminal sialic acid residue and a penultimate β-galactose residue. The modification may involve retargeting the vector by temporarily functionally ablate AAV9 binding in a subset of cells, thereby redirecting the vector to another subset of cells. Alternatively, the modification may involve increasing cellular update efficiency by treating the cells with a neuraminidase to expose cell surface β-galactose. Also provided are compositions containing the AAV9 vector and a neuraminidase. Also provided is a method for purifying AAV9 using β-galactose linked to solid support. Also provided are mutant vectors which have been modified to alter their targeting specificity, including mutant AAV9 in which the galactose binding domain is mutated and AAV in which an AAV9 galactose binding domain is engineered.

    摘要翻译： 描述了改变具有包含AAV9细胞表面结合结构域的衣壳的腺相关病毒（AAV）病毒载体的靶向和/或细胞摄取效率的方法。 该方法包括修饰进化枝F细胞表面受体，其包含具有末端唾液酸残基的聚糖和倒数第二半乳糖残基。 该修饰可能涉及通过临时功能地消融细胞子集中的AAV9结合来重新定向载体，从而将载体重定向到另一个细胞亚群。 或者，修饰可以涉及通过用神经氨酸酶处理细胞以暴露细胞表面β-半乳糖来增加细胞更新效率。 还提供了含有AAV9载体和神经氨酸酶的组合物。 还提供了使用与固体支持物连接的β-半乳糖来纯化AAV9的方法。 还提供已被修饰以改变其靶向特异性的突变载体，包括其中半乳糖结合结构域突变的突变AAV9和其中AAV9半乳糖结合结构域被工程化的AAV。