Adenovirus vectors
    1.
    发明授权
    Adenovirus vectors 有权
    腺病毒载体

    公开(公告)号:US6083750A

    公开(公告)日:2000-07-04

    申请号:US245497

    申请日:1999-02-05

    Abstract: The present invention provides improved adenovirus vectors and packaging cell lines. One type of improved adenoviral vector comprises deletions within the E2b region of the adenoviral genome. These E2b-deleted virus are used in conjunction with novel cell lines that constitutively express E2b gene products. The present invention further provides adenoviral vectors deleted for all viral coding regions. These "gutted" vectors permit the transfer of large genes to cells as demonstrated herein by the transfer of the dystrophin gene to the muscle of mice. The E2b-deleted vectors and the gutted vectors provide improved adenoviral vectors useful for a wide variety of gene therapy applications.

    Abstract translation: 本发明提供了改进的腺病毒载体和包装细胞系。 一种类型的改良的腺病毒载体包含在腺病毒基因组的E2b区域内的缺失。 这些E2b缺失的病毒与组成型表达E2b基因产物的新型细胞系一起使用。 本发明还提供了所有病毒编码区缺失的腺病毒载体。 这些“内含”载体允许将大基因转移到细胞中,如本文通过将肌营养不良蛋白基因转移到小鼠的肌肉所证明的。 E2b缺失的载体和内切的载体提供了可用于各种基因治疗应用的改良的腺病毒载体。

    Helper adenovirus vectors
    3.
    发明授权
    Helper adenovirus vectors 有权
    辅助腺病毒载体

    公开(公告)号:US06451596B1

    公开(公告)日:2002-09-17

    申请号:US09562919

    申请日:2000-05-02

    Abstract: The present invention provides improved adenovirus vectors and packaging cell lines. One type of improved adenoviral vector comprises deletions within the E2b region of the adenoviral genome. These E2b-deleted virus are used in conjunction with novel cell lines that constitutively express E2b gene products. The present invention further provides adenoviral vectors deleted for all viral coding regions. These “gutted” vectors permit the transfer of large genes to cells as demonstrated herein by the transfer of the dystrophin gene to the muscle of mice. The E2b-deleted vectors and the gutted vectors provide improved adenoviral vectors useful for a wide variety of gene therapy applications.

    Abstract translation: 本发明提供了改进的腺病毒载体和包装细胞系。 一种类型的改良的腺病毒载体包含在腺病毒基因组的E2b区域内的缺失。 这些E2b缺失的病毒与组成型表达E2b基因产物的新型细胞系一起使用。 本发明还提供了所有病毒编码区缺失的腺病毒载体。 这些“内含”载体允许将大基因转移到细胞中,如本文通过将肌营养不良蛋白基因转移到小鼠的肌肉所证明的。 E2b缺失的载体和内切的载体提供了可用于各种基因治疗应用的改良的腺病毒载体。

    Production of viral vectors
    4.
    发明授权
    Production of viral vectors 有权
    生产病毒载体

    公开(公告)号:US08637313B2

    公开(公告)日:2014-01-28

    申请号:US12884027

    申请日:2010-09-16

    Abstract: The present invention relates to methods and compositions for the production of viral vectors. In particular, the present invention provides methods and compositions for faster, higher titer and higher purity production of viral vectors (e.g. adenoviral vectors). In some embodiments, the present invention provides gutted and helper viruses with identical or similar termini. In other embodiments, the present invention provides terminal protein linked adenoviral DNA. In certain embodiments, the present invention provides template extended adenoviral DNA.

    Abstract translation: 本发明涉及用于生产病毒载体的方法和组合物。 特别地,本发明提供了用于更快,更高滴定度和更高纯度产生病毒载体(例如腺病毒载体)的方法和组合物。 在一些实施方案中,本发明提供具有相同或相似末端的内切和辅助病毒。 在其它实施方案中,本发明提供了末端蛋白质连接的腺病毒DNA。 在某些实施方案中,本发明提供扩增模板的腺病毒DNA。

    Mini-dystrophin nucleic acid sequences
    5.
    发明授权
    Mini-dystrophin nucleic acid sequences 有权
    迷你抗肌营养不良蛋白核酸序列

    公开(公告)号:US06869777B2

    公开(公告)日:2005-03-22

    申请号:US10149736

    申请日:2001-10-04

    CPC classification number: C07K14/4708 A01K2217/05 A61K48/00 C12N2799/022

    Abstract: The present invention relates to compositions and methods for expressing mini-dystrophin peptides. In particular, the present invention provides compositions comprising nucleic acid sequences that are shorter than wild-type dystrophin cDNA and that express mini-dystrophin peptides that function in a similar manner as wild-type dystrophin proteins. The present invention also provides compositions comprising mini-dystrophin peptides, and methods for expressing mini-dystrophin peptides in target cells.

    Abstract translation: 本发明涉及用于表达微型肌营养不良蛋白肽的组合物和方法。 特别地,本发明提供了包含比野生型肌营养不良蛋白cDNA短的核酸序列并且表达与野生型肌营养不良蛋白类似的功能的微型肌营养不良蛋白肽的组合物。 本发明还提供了包含迷你抗肌营养不良蛋白肽的组合物,以及用于在靶细胞中表达微型肌营养不良蛋白肽的方法。

    Nucleic acid sequences encoding peptides with utrophin spectrin-like repeats
    8.
    发明授权
    Nucleic acid sequences encoding peptides with utrophin spectrin-like repeats 有权
    编码具有utrophin spectrin样重复肽的核酸序列

    公开(公告)号:US08501920B2

    公开(公告)日:2013-08-06

    申请号:US11824870

    申请日:2007-07-03

    CPC classification number: C07K14/4708 A01K2217/05 A61K48/00 C12N2799/022

    Abstract: The present invention relates to compositions and methods for expressing mini-dystrophin peptides. In particular, the present invention provides compositions comprising nucleic acid sequences that are shorter than wild-type dystrophin cDNA and that express mini-dystrophin peptides that function in a similar manner as wild-type dystrophin proteins. The present invention also provides compositions comprising mini-dystrophin peptides, and methods for expressing mini-dystrophin peptides in target cells.

    Abstract translation: 本发明涉及用于表达微型肌营养不良蛋白肽的组合物和方法。 特别地,本发明提供了包含比野生型肌营养不良蛋白cDNA短的核酸序列并且表达与野生型肌营养不良蛋白类似的功能的微型肌营养不良蛋白肽的组合物。 本发明还提供了包含迷你抗肌营养不良蛋白肽的组合物,以及用于在靶细胞中表达微型肌营养不良蛋白肽的方法。

    Production Of Viral Vectors
    9.
    发明申请
    Production Of Viral Vectors 有权
    生产病毒载体

    公开(公告)号:US20110033926A1

    公开(公告)日:2011-02-10

    申请号:US12884027

    申请日:2010-09-16

    Abstract: The present invention relates to methods and compositions for the production of viral vectors. In particular, the present invention provides methods and compositions for faster, higher titer and higher purity production of viral vectors (e.g. adenoviral vectors). In some embodiments, the present invention provides gutted and helper viruses with identical or similar termini. In other embodiments, the present invention provides terminal protein linked adenoviral DNA. In certain embodiments, the present invention provides template extended adenoviral DNA.

    Abstract translation: 本发明涉及用于生产病毒载体的方法和组合物。 特别地,本发明提供了用于更快,更高滴定度和更高纯度产生病毒载体(例如腺病毒载体)的方法和组合物。 在一些实施方案中,本发明提供具有相同或相似末端的内切和辅助病毒。 在其它实施方案中,本发明提供了末端蛋白质连接的腺病毒DNA。 在某些实施方案中,本发明提供扩增模板的腺病毒DNA。

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