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    CAPSID-MODIFIED RAAV VECTOR COMPOSITIONS AND METHODS THEREFOR
    4.
    发明申请
    CAPSID-MODIFIED RAAV VECTOR COMPOSITIONS AND METHODS THEREFOR 有权
    CAPSID改性的RAAV VECTOR组合物及其方法

    公开(公告)号：US20140341852A1

    公开(公告)日：2014-11-20

    申请号：US14214011

    申请日：2014-03-14

    Applicant: University of Florida Research Foundation, Inc.

    Inventor： Arun Srivastava ,  George V. Aslanidi ,  Sergei Zolotukhin ,  Mavis Agbandje-McKenna ,  Kim M. Van Vliet ,  Li Zhong ,  Lakshmanan Govindasamy

    IPC: C07K14/005 ,  C07K14/805 ,  A61K38/42 ,  C12N7/00 ,  C12N15/86

    CPC classification number: C07K14/005 ,  A61K38/42 ,  C07K14/805 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14171 ,  C12N2810/6027

    Abstract: Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations containing them. Also provided are methods of preparing and using the disclosed capsid-protein-mutated rAAV constructs in a variety of diagnostic and therapeutic modalities, including, inter alia, as mammalian cell-targeting delivery agents, and as human gene therapy vectors. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.

    Abstract translation: 公开了衣壳修饰的rAAV表达载体，以及感染性病毒粒子，组合物和含有它们的药物制剂。 还提供了制备和使用所公开的衣壳蛋白突变的rAAV构建体在各种诊断和治疗方式中的方法，所述方法尤其包括作为哺乳动物细胞靶向递送剂，以及作为人基因治疗载体。 还公开了用于衣壳修饰的rAAV表达载体，病毒颗粒和具有相对于未修饰的rAAV载体的转导效率具有改进的转导效率的感染性病毒粒子的大规模生产方法，以及在制造中使用所公开的组合物 的用于各种体外和/或体内应用的药物。

    Capsid-mutated rAAV vectors and methods of use
    6.
    发明授权
    Capsid-mutated rAAV vectors and methods of use 有权

    公开(公告)号：US10426844B2

    公开(公告)日：2019-10-01

    申请号：US15246385

    申请日：2016-08-24

    Applicant: University of Florida Research Foundation, Inc.

    Inventor： Mavis Agbandje-McKenna ,  William W. Hauswirth ,  Arun Srivastava ,  Li Zhong

    IPC: C12N15/86 ,  A61K48/00 ,  C07K14/705 ,  A61K45/06 ,  C07K14/72

    Abstract: Disclosed are capsid-mutated rAAV vectors and methods for their use in gene therapy, and particularly for use in delivering therapeutic transgenes to treat a variety of mammalian diseases and disorders, including dysfunctions and abnormal conditions of the human eye. VP3 capsid proteins comprising a modification of one or more of the surface-exposed tyrosine residues are disclosed, and in particular, VP3 capsid protein comprising tyrosine-to-phenylalanine mutations at positions corresponding to Y444F, Y500F, and Y730F of the wild-type AAV2 sequence. Also provided are rAAV virions and viral particles that comprise such a mutated AAV capsid protein and a nucleic acid molecule that expresses one or more selected therapeutic or reporter transgenes in one or more mammalian cells of interest. Advantageously, the capsid-mutated rAAV vectors and virions disclosed herein afford improved transduction efficiency in a variety of cells, tissues and organs of interest, when compared to their unmodified (i.e., wild-type) rAAV vector counterparts.

    CAPSID-MODIFIED, RAAV3 VECTOR COMPOSITIONS AND USES IN GENE THERAPY OF HUMAN LIVER CANCER
    8.
    发明申请
    CAPSID-MODIFIED, RAAV3 VECTOR COMPOSITIONS AND USES IN GENE THERAPY OF HUMAN LIVER CANCER 审中-公开
    CAPSID改性，RAAV3载体组合物和人类肝癌基因治疗中的用途

    公开(公告)号：US20160333372A1

    公开(公告)日：2016-11-17

    申请号：US14891241

    申请日：2014-05-21

    Applicant: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.

    Inventor： Arun Srivastava ,  Li Zhong ,  Sergei Zolotukhin ,  George Vladimirovich Aslanidi ,  Mavis Agbandje-McKenna ,  Kim M. Van Vliet

    IPC: C12N15/86

    Abstract: Disclosed are next-generation multi-mutated capsid protein-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using these high transduction efficiency vector constructs in a variety of therapeutic applications including, inter alia, as delivery agents for the treatment or amelioration of one or more diseases or abnormal conditions in an affected mammal using in vivo and/or ex situ viral vector-based gene therapy protocols. Also disclosed are large-scale production methods for the multi-mutated, capsid-modified rAAV expression vectors, viral particles, and infectious virions, as well as use of the disclosed compositions in the manufacture of medicaments for use in a variety of in vitro and/or in vivo therapeutic methodologies.

    Abstract translation: 公开了下一代多突变衣壳蛋白修饰的rAAV表达载体，以及包含它们的感染性病毒粒子，组合物和药物制剂。 还公开了在各种治疗应用中制备和使用这些高转导效率载体构建体的方法，包括特别地，作为在受影响的哺乳动物中使用体内和/或体内治疗或改善一种或多种疾病或异常状况的递送剂，和/ 或基于病毒载体的基因治疗方案。 还公开了用于多突变的衣壳修饰的rAAV表达载体，病毒颗粒和感染性病毒粒子的大规模生产方法，以及所公开的组合物在制备用于各种体外和 /或体内治疗方法。

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