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    Modified virus vectors and methods of making and using the same
    32.
    发明授权
    Modified virus vectors and methods of making and using the same 有权
    修饰病毒载体及其制备和使用方法

    公开(公告)号：US09475845B2

    公开(公告)日：2016-10-25

    申请号：US14543459

    申请日：2014-11-17

    Applicant: The University of North Carolina at Chapel Hill

    Inventor： Aravind Asokan ,  Richard Jude Samulski

    IPC: C07K14/005 ,  C12N15/86

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

    Abstract: The present invention provides AAV capsid proteins (VP1, VP2 and/or VP3) comprising a modification in the amino acid sequence in the three-fold axis loop 4 and virus capsids and virus vectors comprising the modified AAV capsid protein. In particular embodiments, the modification comprises a substitution of one or more amino acids at amino acid positions 585 to 590 (inclusive) of the native AAV2 capsid protein sequence or the corresponding positions of other AAV capsid proteins. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject in vivo.

    Abstract translation: 本发明提供了包含三折轴环4中的氨基酸序列修饰的AAV衣壳蛋白（VP1，VP2和/或VP3）以及包含经修饰的AAV衣壳蛋白的病毒衣壳和病毒载体。 在具体实施方案中，修饰包括在天然AAV2衣壳蛋白序列的585至590（含）位置的一个或多个氨基酸取代或其它AAV衣壳蛋白的相应位置。 本发明还提供了将本发明的病毒载体和病毒衣壳体内给予细胞或受试者的方法。

    Mutant adeno-associated virus virions and methods of use thereof
    33.
    发明授权
    Mutant adeno-associated virus virions and methods of use thereof 有权
    突变型腺相关病毒颗粒及其使用方法

    公开(公告)号：US09233131B2

    公开(公告)日：2016-01-12

    申请号：US12277164

    申请日：2008-11-24

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

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

    IPC: C12N15/861 ,  A61K39/12 ,  A61K35/76 ,  A61K31/7088 ,  A61K38/17 ,  C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  A61K48/00

    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。

    CAPSID-MODIFIED RAAV VECTOR COMPOSITIONS AND METHODS THEREFOR
    36.
    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。

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