利索-全球专利检索

  1. 登录
  2. 注册

搜索结果

13 条记录 (耗时 0.019 秒)

    Raav expression systems for genetic modification of specific capsid proteins
    1.
    发明申请
    Raav expression systems for genetic modification of specific capsid proteins 有权
    用于特异性衣壳蛋白遗传修饰的Raav表达系统

    公开(公告)号：US20060088936A1

    公开(公告)日：2006-04-27

    申请号：US10513059

    申请日：2003-05-01

    申请人： Kenneth Warrington ,  Shaun Opie ,  Nicholas Muzyczka

    发明人： Kenneth Warrington ,  Shaun Opie ,  Nicholas Muzyczka

    IPC分类号： C12N15/861

    CPC分类号： A61K48/005 ,  C07K14/005 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/50 ,  C12N2810/858

    摘要： Disclosed are improved recombinant adeno-associated viral (rAAV) vectors having mutations in one or more capsid proteins. Exemplary vectors are provided that have altered affinity for heparin or heparin sulfate, as well as vectors, expression systems, and rAAV virions that lack functional VP2 protein expression, but are nevertheless, fully virulent. Also provided by the invention are rAAV vector-based compositions, virus particles, host cells, and pharmaceutical formulations that comprise them useful in the expression of selected therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in selected mammals, including organs, tissues, and human host cells.

    摘要翻译： 公开了在一种或多种衣壳蛋白中具有突变的改良的重组腺相关病毒（rAAV）载体。 提供了对肝素或硫酸肝素具有改变的亲和力的示例性载体，以及缺乏功能性VP2蛋白表达但仍然完全毒性的载体，表达系统和rAAV病毒体。 本发明还提供了基于rAAV载体的组合物，病毒颗粒，宿主细胞和药物制剂，其包含它们可用于选择的哺乳动物（包括器官）中选择的治疗性蛋白质，多肽，肽，反义寡核苷酸和/或核酶的表达， 组织和人宿主细胞。

    Raav expression systems for genetic modification of specific capsid proteins
    3.
    发明授权
    Raav expression systems for genetic modification of specific capsid proteins 有权
    用于特异性衣壳蛋白遗传修饰的Raav表达系统

    公开(公告)号：US08802080B2

    公开(公告)日：2014-08-12

    申请号：US10513059

    申请日：2003-05-01

    申请人： Kenneth H. Warrington ,  Shaun R. Opie ,  Nicholas Muzyczka

    发明人： Kenneth H. Warrington ,  Shaun R. Opie ,  Nicholas Muzyczka

    IPC分类号： C12N15/864 ,  C12N7/01 ,  C12N5/10 ,  C12N15/63 ,  C07K14/015

    CPC分类号： A61K48/005 ,  C07K14/005 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/50 ,  C12N2810/858

    摘要： Disclosed are improved recombinant adeno-associated viral (rAAV) vectors having mutations in one or more capsid proteins. Exemplary vectors are provided that have altered affinity for heparin or heparin sulfate, as well as vectors, expression systems, and rAAV virions that lack functional VP2 protein expression, but are nevertheless, fully virulent. Also provided by the invention are rAAV vector-based compositions, virus particles, host cells, and pharmaceutical formulations that comprise them useful in the expression of selected therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in selected mammals, including organs, tissues, and human host cells.

    摘要翻译： 公开了在一种或多种衣壳蛋白中具有突变的改良的重组腺相关病毒（rAAV）载体。 提供了对肝素或硫酸肝素具有改变的亲和力的示例性载体，以及缺乏功能性VP2蛋白表达但仍然是完全毒性的载体，表达系统和rAAV病毒体。 本发明还提供了基于rAAV载体的组合物，病毒颗粒，宿主细胞和药物制剂，其包含它们可用于选择的哺乳动物（包括器官）中选择的治疗性蛋白质，多肽，肽，反义寡核苷酸和/或核酶的表达， 组织和人宿主细胞。

    METHODS OF GENE DELIVERY USING CAPSID-MODIFIED RAAV EXPRESSION SYSTEMS
    5.
    发明申请
    METHODS OF GENE DELIVERY USING CAPSID-MODIFIED RAAV EXPRESSION SYSTEMS 审中-公开
    使用CAPSID修改RAAV表达系统的基因交付方法

    公开(公告)号：US20150005369A1

    公开(公告)日：2015-01-01

    申请号：US14326293

    申请日：2014-07-08

    申请人： University of Florida Research Foundation, Inc.

    发明人： Nicholas Muzyczka ,  Shaun R. Opie ,  Kenneth H. Warrington

    IPC分类号： A61K48/00

    CPC分类号： A61K48/005 ,  C07K14/005 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/50 ,  C12N2810/858

    摘要： Disclosed are methods of gene delivery using capsid-modified recombinant adeno-associated viral (rAAV) vectors. Exemplary methods are provided employing vectors that have altered affinity for heparin or heparin sulfate, as well as vectors, expression systems, and rAAV virions that lack functional VP2 protein expression, but are nevertheless, fully virulent. Also provided by the invention are methods employing the rAAV vector-based compositions, virus particles, host cells, and pharmaceutical formulations in the expression of selected therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in selected mammals, including organs, tissues, and human host cells.

    摘要翻译： 公开了使用衣壳修饰的重组腺相关病毒（rAAV）载体的基因递送方法。 使用对肝素或硫酸肝素具有改变的亲和力的载体以及缺乏功能性VP2蛋白表达但仍具有完全毒性的载体，表达系统和rAAV病毒粒子提供了示例性的方法。 本发明还提供了在所选择的哺乳动物（包括器官，组织）中选择的治疗性蛋白质，多肽，肽，反义寡核苷酸和/或核酶的表达中使用基于rAAV载体的组合物，病毒颗粒，宿主细胞和药物制剂的方法 ，和人宿主细胞。

    Improved rAAv vectors
    6.
    发明申请
    Improved rAAv vectors 审中-公开
    改进的rAAv载体

    公开(公告)号：US20060292117A1

    公开(公告)日：2006-12-28

    申请号：US10511914

    申请日：2003-04-17

    申请人： Scott Loiler ,  Terence Flotte ,  Nicholas Muzyczka ,  Mark Atkinson

    发明人： Scott Loiler ,  Terence Flotte ,  Nicholas Muzyczka ,  Mark Atkinson

    IPC分类号： A61K48/00 ,  A61K9/127 ,  C12N15/861

    CPC分类号： C12N15/86 ,  A61K48/00 ,  C07K14/005 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2810/858

    摘要： Disclosed are methods for the use of therapeutic polypeptide-encoding polynucleotides in the creation of transformed host cells and transgenic animals. In particular, the use of recombinant adeno-associated viral (rAAV) vector compositions that specifically target mammalian cells, such as pancreatic islets cells, that express low-density lipoprotein receptors on their cell surface. The disclosed vectors comprise one or more polynucleotide sequences that express one or more mammalian polypeptides having therapeutic efficacy in the amelioration, treatment and/or prevention of AAT- or cytokine polypeptide deficiencies, such as for example in diabetes and related diseases, as well as a variety of autoimmune disorders including, for example, lupus and rheumatoid arthritis.

    摘要翻译： 公开了在产生转化的宿主细胞和转基因动物中使用治疗性多肽编码多核苷酸的方法。 特别地，使用特异性靶向哺乳动物细胞的重组腺相关病毒（rAAV）载体组合物，所述哺乳动物细胞例如胰岛细胞在其细胞表面上表达低密度脂蛋白受体。 所公开的载体包含一种或多种多核苷酸序列，其表达一种或多种在改善，治疗和/或预防AAT或细胞因子多肽缺陷（例如糖尿病和相关疾病）中具有治疗功效的哺乳动物多肽，以及 多种自身免疫性疾病，包括例如狼疮和类风湿性关节炎。