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    Adenoviral vector with replication-dependent transgene expression
    5.
    发明申请
    Adenoviral vector with replication-dependent transgene expression 有权
    具有复制依赖性转基因表达的腺病毒载体

    公开(公告)号：US20050063953A1

    公开(公告)日：2005-03-24

    申请号：US10911957

    申请日：2004-08-05

    申请人： Erik Falck-Pedersen ,  Jason Gall

    发明人： Erik Falck-Pedersen ,  Jason Gall

    IPC分类号： A61K48/00 ,  C12N15/861

    CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N2710/10343 ,  C12N2840/44

    摘要： The invention provides an adenoviral vector comprising (a) at least a portion of an adenoviral genome comprising a major late transcription unit containing a terminal exon, wherein the terminal exon comprises a 5′ splice acceptor DNA sequence element and a 3′ polyadenylation signal sequence, and (b) a non-native nucleic acid sequence encoding a protein that does not contribute to the adenoviral vector entry into a host cell, wherein the non-native nucleic acid sequence is positioned within the terminal exon, such that the non-native nucleic acid sequence is selectively expressed in cells within which the adenoviral vector can replicate. The invention further provides an adenoviral vector composition and a method for treating or preventing a pathologic state in a mammal, comprising administering to the mammal the adenoviral vector composition of the invention in an amount sufficient to treat or prevent the pathologic state in the mammal.

    摘要翻译： 本发明提供了一种腺病毒载体，其包含（a）至少一部分腺病毒基因组，其包含含有末端外显子的主要晚期转录单位，其中所述末端外显子包含5'剪接受体DNA序列元件和3'多腺苷酸化信号序列， 和（b）编码不对腺病毒载体进入宿主细胞有贡献的蛋白质的非天然核酸序列，其中非天然核酸序列位于末端外显子内，使得非天然核酸 酸序列在腺病毒载体可以复制的细胞中选择性表达。 本发明进一步提供了一种用于治疗或预防哺乳动物病理状态的腺病毒载体组合物和方法，其包括以足以治疗或预防哺乳动物病理状态的量向哺乳动物施用本发明的腺病毒载体组合物。

    Use of AAV integration efficiency element for mediating site-specific integration of a transcription unit
    6.
    发明申请
    Use of AAV integration efficiency element for mediating site-specific integration of a transcription unit 审中-公开
    使用AAV整合效率元素来调节转录单位的位点特异性整合

    公开(公告)号：US20050106125A1

    公开(公告)日：2005-05-19

    申请号：US10959017

    申请日：2004-10-05

    申请人： Erik Falck-Pedersen ,  Nicola Philpott

    发明人： Erik Falck-Pedersen ,  Nicola Philpott

    IPC分类号： A61K48/00 ,  C12N15/861 ,  C12N15/864 ,  C12N15/90

    CPC分类号： C12N15/86 ,  A61K48/00 ,  C07K14/005 ,  C12N15/90 ,  C12N2710/10343 ,  C12N2710/10344 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2800/30

    摘要： The invention provides an expression construct comprising a nucleic acid sequence encoding an adeno-associated virus integration efficiency element (AAV IEE), wherein the expression construct is substantially devoid of AAV inverted terminal repeats (AAV ITRs). Such an expression construct site-specifically integrates into a host cell chromosome when provided to a host cell in conjunction with an AAV Rep protein. The invention also provides a method of integrating a nucleic acid sequence of interest into a host cell chromosome through use of such an expression construct, as well as a method of prophylactically or therapeutically treating a mammal for a pathologic state comprising administering to a mammal such an expression construct comprising a nucleic acid sequence encoding a therapeutic factor.

    摘要翻译： 本发明提供了包含编码腺相关病毒整合效率元件（AAV IEE）的核酸序列的表达构建体，其中所述表达构建体基本上没有AAV反向末端重复序列（AAV ITR）。 当与AAV Rep蛋白结合提供给宿主细胞时，这种表达构建位点特异性地整合到宿主细胞染色体中。 本发明还提供了通过使用这种表达构建体将感兴趣的核酸序列整合到宿主细胞染色体中的方法，以及预防或治疗哺乳动物治疗病理状态的方法，包括向哺乳动物施用 表达构建体，其包含编码治疗因子的核酸序列。