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    Tissue specific gene therapy treatment
    1.
    发明授权
    Tissue specific gene therapy treatment 有权
    组织特异性基因治疗

    公开(公告)号：US08383601B2

    公开(公告)日：2013-02-26

    申请号：US12447558

    申请日：2007-10-30

    申请人： Walter J. Koch ,  Patrick Most ,  Sven T. Pleger ,  Joseph E. Rabinowitz

    发明人： Walter J. Koch ,  Patrick Most ,  Sven T. Pleger ,  Joseph E. Rabinowitz

    IPC分类号： A61K48/00 ,  C12N15/11 ,  C12N5/00

    CPC分类号： C12N15/86 ,  C07K14/4728 ,  C12N2750/14143 ,  C12N2830/008

    摘要： The invention provides nucleic acid segments, compositions and methods for the treatment of heart failure, vascular dysfunction, endothelial dysfunction, diabetes, [Ca2+]i regulation and NO synthase dysfunction. Adeno-associated and adenovirus are used as gene delivery vectors for the nucleic acid segments to product long term over-expression of S100A1, a small calcium sensing protein associated with the disclosed ailments and dysfunctions.

    摘要翻译： 本发明提供用于治疗心力衰竭，血管功能障碍，内皮功能障碍，糖尿病，[Ca 2+] i调节和NO合成酶功能障碍的核酸区段，组合物和方法。 腺相关腺病毒和腺病毒用作核酸片段的基因递送载体以产生S100A1的长期过表达，S100A1是与所公开的疾病和功能障碍相关的小的钙感觉蛋白。

    TISSUE SPECIFIC GENE THERAPY TREATMENT
    2.
    发明申请
    TISSUE SPECIFIC GENE THERAPY TREATMENT 有权
    组织特异性基因治疗

    公开(公告)号：US20100190840A1

    公开(公告)日：2010-07-29

    申请号：US12447558

    申请日：2007-10-30

    申请人： Walter J. Koch ,  Patrick Most ,  Sven T. Pleger ,  Joseph E. Rabinowitz

    发明人： Walter J. Koch ,  Patrick Most ,  Sven T. Pleger ,  Joseph E. Rabinowitz

    IPC分类号： A61K31/711 ,  C07H21/04 ,  A61P9/04 ,  C12N15/63

    CPC分类号： C12N15/86 ,  C07K14/4728 ,  C12N2750/14143 ,  C12N2830/008

    摘要： The invention provides nucleic acid segments, compositions and methods for the treatment of heart failure, vascular dysfunction, endothelial dysfunction, diabetes, [Ca2+]i regulation and NO synthase dysfunction. Adeno-associated and adenovirus are used as gene delivery vectors for the nucleic acid segments to product long term over-expression of S100A1, a small calcium sensing protein associated with the disclosed ailments and dysfunctions.

    摘要翻译： 本发明提供用于治疗心力衰竭，血管功能障碍，内皮功能障碍，糖尿病，[Ca 2+] i调节和NO合成酶功能障碍的核酸区段，组合物和方法。 腺相关腺病毒和腺病毒用作核酸片段的基因递送载体以产生S100A1的长期过表达，S100A1是与所公开的疾病和功能障碍相关的小的钙感觉蛋白。

    Chimeric Vectors
    4.
    发明申请
    Chimeric Vectors 有权
    嵌合载体

    公开(公告)号：US20110104119A1

    公开(公告)日：2011-05-05

    申请号：US12983547

    申请日：2011-01-03

    申请人： Dawn E. Bowles ,  Chengwen Li ,  Joseph E. Rabinowitz ,  Josh Grieger ,  Mavis Agbandje-McKenna ,  Richard Jude Samulski

    发明人： Dawn E. Bowles ,  Chengwen Li ,  Joseph E. Rabinowitz ,  Josh Grieger ,  Mavis Agbandje-McKenna ,  Richard Jude Samulski

    IPC分类号： A61K35/76 ,  C12N5/00 ,  A61P21/00 ,  A61P3/10 ,  A61P9/00 ,  A61P35/00 ,  A61P27/02

    CPC分类号： C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14144

    摘要： The present invention is based, in part, on the discovery that parvovirus (including AAV) capsids can be engineered to incorporate small, selective regions from other parvoviruses that confer desirable properties. The inventors have discovered that in some cases as little as a single amino acid insertion or substitution from a first parvovirus (e.g., an AAV) into the capsid structure of another parvovirus (e.g., an AAV) to create a chimeric parvovirus is sufficient to confer one or more of the desirable properties of the first parvovirus to the resulting chimeric parvovirus and/or to confer a property that is not exhibited by the first parvovirus or is present to a lesser extent.

    摘要翻译： 本发明部分地基于发现细小病毒（包括AAV）衣壳可以被工程化以包含来自其他细小病毒的小的选择性区域，其赋予期望的性质。 本发明人已经发现，在一些情况下，只要将单一氨基酸插入或从第一细小病毒（例如，AAV）置换到另一种细小病毒（例如AAV）的衣壳结构中以产生嵌合细小病毒就足够了 第一细小病毒对所得嵌合细小病毒的一个或多个所需性质和/或赋予第一细小病毒不表现的性质或者以较小程度存在的性质。

    Chimeric vectors
    6.
    发明授权
    Chimeric vectors 有权
    嵌合载体

    公开(公告)号：US09012224B2

    公开(公告)日：2015-04-21

    申请号：US12983547

    申请日：2011-01-03

    申请人： Dawn E. Bowles ,  Chengwen Li ,  Joseph E. Rabinowitz ,  Josh Grieger ,  Mavis Agbandje-McKenna ,  Richard Jude Samulski

    发明人： Dawn E. Bowles ,  Chengwen Li ,  Joseph E. Rabinowitz ,  Josh Grieger ,  Mavis Agbandje-McKenna ,  Richard Jude Samulski

    IPC分类号： C12N15/861 ,  C12N15/86

    CPC分类号： C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14144

    摘要： The present invention is based, in part, on the discovery that parvovirus (including AAV) capsids can be engineered to incorporate small, selective regions from other parvoviruses that confer desirable properties. The inventors have discovered that in some cases as little as a single amino acid insertion or substitution from a first parvovirus (e.g., an AAV) into the capsid structure of another parvovirus (e.g., an AAV) to create a chimeric parvovirus is sufficient to confer one or more of the desirable properties of the first parvovirus to the resulting chimeric parvovirus and/or to confer a property that is not exhibited by the first parvovirus or is present to a lesser extent.

    摘要翻译： 本发明部分地基于发现细小病毒（包括AAV）衣壳可以被工程化以包含来自其他细小病毒的小的选择性区域，其赋予期望的性质。 本发明人已经发现，在一些情况下，只要将单一氨基酸插入或从第一细小病毒（例如，AAV）置换到另一种细小病毒（例如AAV）的衣壳结构中以产生嵌合细小病毒就足够了 第一细小病毒对所得嵌合细小病毒的一个或多个所需性质和/或赋予第一细小病毒不表现的性质或者以较小程度存在的性质。

    Chimeric vectors
    7.
    发明授权
    Chimeric vectors 有权
    嵌合载体

    公开(公告)号：US07892809B2

    公开(公告)日：2011-02-22

    申请号：US11793430

    申请日：2005-12-15

    申请人： Dawn E. Bowles ,  Chengwen Li ,  Joseph E. Rabinowitz ,  Josh Grieger ,  Mavis Agbandje-McKenna ,  Richard Jude Samulski

    发明人： Dawn E. Bowles ,  Chengwen Li ,  Joseph E. Rabinowitz ,  Josh Grieger ,  Mavis Agbandje-McKenna ,  Richard Jude Samulski

    IPC分类号： C12N7/01

    CPC分类号： C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14144

    摘要： The present invention is based, in part, on the discovery that parvovirus (including AAV) capsids can be engineered to incorporate small, selective regions from other parvoviruses that confer desirable properties. The inventors have discovered that in some cases as little as a single amino acid insertion or substitution from a first parvovirus (e.g., an AAV) into the capsid structure of another parvovirus (e.g., an AAV) to create a chimeric parvovirus is sufficient to confer one or more of the desirable properties of the first parvovirus to the resulting chimeric parvovirus and/or to confer a property that is not exhibited by the first parvovirus or is present to a lesser extent.

    摘要翻译： 本发明部分地基于发现细小病毒（包括AAV）衣壳可以被工程化以包含来自其他细小病毒的小的选择性区域，其赋予期望的性质。 本发明人已经发现，在一些情况下，只要将单一氨基酸插入或从第一细小病毒（例如，AAV）置换到另一种细小病毒（例如AAV）的衣壳结构中以产生嵌合细小病毒就足够了 第一细小病毒对所得嵌合细小病毒的一个或多个所需性质和/或赋予第一细小病毒不表现的性质或者以较小程度存在的性质。

    CONSTRUCTS AND METHODS FOR DELIVERING MOLECULES VIA VIRAL VECTORS WITH BLUNTED INNATE IMMUNE RESPONSES
    10.
    发明申请
    CONSTRUCTS AND METHODS FOR DELIVERING MOLECULES VIA VIRAL VECTORS WITH BLUNTED INNATE IMMUNE RESPONSES 审中-公开
    通过流行的免疫反应的病毒载体递送分子的构建和方法

    公开(公告)号：US20160222414A1

    公开(公告)日：2016-08-04

    申请号：US15099651

    申请日：2016-04-15

    申请人： Joseph E. Rabinowitz ,  The Trustees of the University of Pennsylvania

    发明人： Joseph E. Rabinowitz ,  James M. Wilson ,  Susan M. Faust

    IPC分类号： C12N15/86 ,  A61K48/00 ,  C12N7/00

    CPC分类号： C12N15/86 ,  A61K48/0058 ,  A61K48/0075 ,  A61K48/0091 ,  C12N7/00 ,  C12N2750/14141 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2800/24

    摘要： A CpG-modified recombinant adeno-associated viral (AAV) vector is described. The vector carries a nucleic acid molecule comprising AAV inverted terminal repeat (ITR) sequences and an exogenous gene sequence under the control of regulatory sequences which control expression of the gene product, in which the nucleic acid sequences carried by the vector are modified to significantly reduce CpG di-nucleotides such that an immune response to the vector is reduced as compared to the unmodified AAV vector. Also provided are methods and regimens for delivering transgenes using these AAV viral vectors, in which the innate immune response to the vector and/or transgene is significantly modulated.

    摘要翻译： 描述了CpG修饰的重组腺相关病毒（AAV）载体。 载体携带包含AAV反向末端重复序列（ITR）序列的核酸分子和外源基因序列，其在控制基因产物表达的调节序列的控制下，其中载体携带的核酸序列被修饰以显着降低 CpG二核苷酸，使得与未修饰的AAV载体相比，对载体的免疫应答降低。 还提供了使用这些AAV病毒载体递送转基因的方法和方案，其中对载体和/或转基因的先天免疫应答被显着调节。