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    CAPSID-MODIFIED, RAAV3 VECTOR COMPOSITIONS AND USES IN GENE THERAPY OF HUMAN LIVER CANCER
    1.
    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。

    HIGH-TRANSDUCTION-EFFICIENCY RAAV VECTORS, COMPOSITIONS, AND METHODS OF USE
    6.
    发明申请
    HIGH-TRANSDUCTION-EFFICIENCY RAAV VECTORS, COMPOSITIONS, AND METHODS OF USE 有权
    高传导效率RAAV矢量，组成和使用方法

    公开(公告)号：US20150133530A1

    公开(公告)日：2015-05-14

    申请号：US14401442

    申请日：2013-05-15

    Applicant: University of Florida Research Foundation, Inc.

    Inventor： Arun Srivastava ,  George Vladimirovich Aslanidi ,  Kim M. Van Vliet ,  Mavis Agbandje-Mckenna

    IPC: C07K14/005 ,  C12N15/86

    CPC classification number: C07K14/005 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14171

    Abstract: The present invention provides AAV capsid proteins comprising modification of one or a combination of the surface-exposed lysine, serine, threonine and/or tyrosine residues in the VP3 region. Also provided are rAAV virions comprising the AAV capsid proteins of the present invention, as well as nucleic acid molecules and rAAV vectors encoding the AAV capsid proteins of the present invention. Advantageously, the rAAV vectors and virions of the present invention have improved efficiency in transduction of a variety of cells, tissues and organs of interest, when compared to wild-type rAAV vectors and virions.

    Abstract translation: 本发明提供AAV衣壳蛋白，其包括VP3区域中的一种或其表面暴露的赖氨酸，丝氨酸，苏氨酸和/或酪氨酸残基的组合的修饰。 还提供了包含本发明的AAV衣壳蛋白的rAAV病毒粒子，以及编码本发明的AAV衣壳蛋白的核酸分子和rAAV载体。 有利的是，当与野生型rAAV载体和病毒粒子相比时，本发明的rAAV载体和病毒粒子改善了感兴趣的各种细胞，组织和器官的转导效率。

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