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    IMPROVED RAAV VECTORS AND METHODS FOR TRANSDUCTION OF PHOTORECEPTORS AND RPE CELLS
    52.
    发明申请
    IMPROVED RAAV VECTORS AND METHODS FOR TRANSDUCTION OF PHOTORECEPTORS AND RPE CELLS 审中-公开
    改进的RAAV矢量和转移光电子和RPE细胞的方法

    公开(公告)号：US20160369299A1

    公开(公告)日：2016-12-22

    申请号：US15123515

    申请日：2015-03-04

    Applicant: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.

    Inventor： Shannon E. Boye ,  Sanford L. Boye ,  Mavis Agbandje-McKenna ,  Arun Srivastava

    IPC: C12N15/86 ,  C07K14/005

    CPC classification number: C12N15/86 ,  A61K9/0048 ,  A61K9/5184 ,  A61K48/00 ,  C07K14/005 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2810/6027

    Abstract: Disclosed are capsid-modified rAAV particles and expression vectors, as well as compositions and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and using novel capsid-protein-mutated particle or rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian eye. Also disclosed are methods for subretinal delivery of therapeutic gene constructs to mammalian photoreceptors and retinal pigment epithelial cells, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of a variety of inherited retinal diseases.

    Abstract translation: 公开了衣壳修饰的rAAV颗粒和表达载体，以及包含它们的组合物和药物制剂。 还公开了在各种诊断和治疗应用中制备和使用新的衣壳蛋白突变的颗粒或rAAV载体构建体的方法，包括尤其用作用于诊断，治疗或改善一种或多种疾病，病症， 或哺乳动物眼睛的功能障碍。 还公开了将治疗性基因构建体视网膜上递送给哺乳动物感光细胞和视网膜色素上皮细胞的方法，以及所公开的组合物在制备用于各种体外和/或体内应用的药物中的用途，包括治疗 各种遗传性视网膜疾病。

    Mutant Adeno-Associated Virus Virions and Methods of Use Thereof
    54.
    发明申请
    Mutant Adeno-Associated Virus Virions and Methods of Use Thereof 审中-公开
    突变型腺相关病毒病毒及其使用方法

    公开(公告)号：US20150132262A1

    公开(公告)日：2015-05-14

    申请号：US14444347

    申请日：2014-07-28

    Applicant: The Regents of the University of California

    Inventor： David V. Schaffer ,  Brian Kaspar ,  Narendra Maheshri

    IPC: A61K35/76 ,  C12N7/00

    CPC classification number: A61K35/76 ,  A61K31/7088 ,  A61K38/1709 ,  A61K48/00 ,  C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14123 ,  C12N2750/14132 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14151 ,  C12N2750/14161 ,  C12N2810/6027 ,  A61K2300/00

    Abstract: The present invention provides mutant adeno-associated virus (AAV) that exhibit altered capsid properties, e.g., reduced binding to neutralizing antibodies in serum and/or altered heparin binding and/or altered infectivity of particular cell types. The present invention further provides libraries of mutant AAV comprising one or more mutations in a capsid gene. The present invention further provides methods of generating the mutant AAV and mutant AAV libraries, and compositions comprising the mutant AAV. The present invention further provides recombinant AAV (rAAV) virions that comprise a mutant capsid protein. The present invention further provides nucleic acids comprising nucleotide sequences that encode mutant capsid proteins, and host cells comprising the nucleic acids. The present invention further provides methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.

    Abstract translation: 本发明提供突变型腺相关病毒（AAV），其显示改变的衣壳性质，例如减少与血清中的中和抗体的结合和/或肝素结合的变化和/或特定细胞类型的改变的感染性。 本发明还提供了在衣壳基因中包含一个或多个突变的突变AAV的文库。 本发明还提供了产生突变AAV和突变AAV文库的方法，以及包含突变体AAV的组合物。 本发明还提供了包含突变型衣壳蛋白的重组AAV（rAAV）病毒粒子。 本发明还提供了包含编码突变衣壳蛋白的核苷酸序列的核酸和包含核酸的宿主细胞。 本发明进一步提供将基因产物递送给个体的方法，所述方法通常涉及向有需要的个体施用有效量的受试者rAAV病毒粒子。

    Compositions and Methods for Altering Tissue Specificity and Improving AAV9-Mediated Gene Transfer
    56.
    发明申请
    Compositions and Methods for Altering Tissue Specificity and Improving AAV9-Mediated Gene Transfer 有权
    改变组织特异性和改善AAV9介导的基因转移的组合物和方法

    公开(公告)号：US20130323226A1

    公开(公告)日：2013-12-05

    申请号：US13985630

    申请日：2012-02-17

    Applicant: James M. Wilson ,  Christie L. Bell ,  Luc H. Vandenberghe

    Inventor： James M. Wilson ,  Christie L. Bell ,  Luc H. Vandenberghe

    IPC: A61K31/7088 ,  A61K31/685 ,  A61K38/47

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

    Abstract: 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.

    Abstract translation: 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。

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