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1. 发明申请

WO2002092786A2 A HELPER DEPENDENT ADENOVIRAL VECTOR SYSTEM AND METHODS FOR USING THE SAME 审中-公开
标题翻译：辅助依赖性腺病毒载体系统及其使用方法
公开(公告)号：WO2002092786A2
公开(公告)日：2002-11-21
申请号：PCT/US2002/009125
申请日：2002-03-25
申请人： THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY , EHRHARDT, Anja , KAY, Mark
发明人： EHRHARDT, Anja , KAY, Mark
IPC分类号： C12N
CPC分类号： C12N15/86 , A61K39/00 , C12N15/90 , C12N2710/10343 , C12N2800/108 , C12N2800/30 , C12N2800/80 , C12N2800/90 , C12N2830/002 , C12N2830/008 , C12N2830/38 , C12N2830/46
摘要： A helper dependent adenoviral vector system is provided. The subject helper dependent adenoviral vector system is made up of: (1) a "gutless" adenoviral vector that include cis-acting human stuffer DNA that provides for in vivo long term, high level expression of a coding sequence present on the vector; (2) an adenoviral helper vector that is characterized by having an adenoviral genome region flanked by recombinase recognition sites, where the helper vectors further include a non-mammalian endonuclease recognition site positioned outside of the adenoviral genome region; and (3) a mammalian cell that expresses the corresponding recombinase and endonuclease, as well as the adenoviral preterminal and polymerase proteins. Also provided are methods of using the subject systems to produce virions having the subject helper dependent adenoviral vectors encapsulated in an adenoviral capsid. In addition, kits for use in practicing the subject methods are provided.
摘要翻译：提供辅助性依赖性腺病毒载体系统。受试者辅助因子依赖性腺病毒载体系统由以下部分组成：（1）“无痛”腺病毒载体，其包括顺式作用人填充DNA，其提供载体上存在的编码序列的体内长期高水平表达; （2）腺病毒辅助载体，其特征在于具有侧翼为重组酶识别位点的腺病毒基因组区域，其中辅助载体还包括位于腺病毒基因组区域外部的非哺乳动物内切核酸酶识别位点; 和（3）表达相应的重组酶和内切核酸酶的哺乳动物细胞，以及腺病毒早产和聚合酶蛋白。还提供了使用受试系统产生具有包封在腺病毒衣壳中的受试者辅助性依赖性腺病毒载体的病毒粒子的方法。此外，还提供了用于实践主题方法的套件。

2. 发明申请

WO2002066611A2 MINIMAL PLASMID VECTORS THAT PROVIDE FOR PERSISTENT AND HIGH LEVEL GENE EXPRESSION AND METHODS FOR USING THE SAME 审中-公开
标题翻译：提供持久性和高水平基因表达的最小等离子体载体及其使用方法
公开(公告)号：WO2002066611A2
公开(公告)日：2002-08-29
申请号：PCT/US2002/004975
申请日：2002-02-15
申请人： THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY , KAY, Mark , NAKAI, Hiroyuki
发明人： KAY, Mark , NAKAI, Hiroyuki
IPC分类号： C12N
CPC分类号： C12N15/85 , C12N2800/90 , C12N2830/008 , C12N2830/15 , C12N2830/42 , C12N2830/85
摘要： Methods are provided for the in vivo introduction of an expression cassette into a target cell of a vascularized organism, e.g., a mammal, in a manner such that the encoded protein of the introduced expression cassette is persistently expressed at a high level in the target cell. In the subject methods, an aqueous formulation of a minimal plasmid vector that includes the expression cassette is administered into the vascular system of the organism. The minimal plasmid vector employed in the subject methods is one that provides for persistent and high level expression of an expression cassette that is present on the vector in a manner that is substantially expression cassette sequence and direction independent. Also provided are the minimal plasmid vectors employed in the subject methods. The subject methods and compositions find use in a variety of different applications, including both research and therapeutic applications, and are particularly suited for use in the in vivo delivery of nucleic acids encoding protein products, particularly where persistent, high level protein expression is desired without integration of the vector into the host genome.
摘要翻译：提供了用于将表达盒体内引入血管生物体例如哺乳动物的靶细胞的方法，使得所导入的表达盒的编码蛋白质在靶细胞中以高水平持续表达。在主题方法中，将包含表达盒的最小质粒载体的水性制剂施用于生物体的血管系统。在本发明方法中使用的最小质粒载体是以基本上表达盒序列和方向独立的方式提供存在于载体上的表达盒的持续和高水平表达的质粒载体。还提供了本发明方法中使用的最小质粒载体。主题方法和组合物可用于各种不同的应用，包括研究和治疗应用，并且特别适用于体内递送编码蛋白质产物的核酸，特别是在需要持久性高水平蛋白质表达而没有将载体整合到宿主基因组中。

3. 发明申请

WO2003010180A1 METHODS AND COMPOSITIONS FOR RNAI MEDIATED INHIBITION OF GENE EXPRESSION IN MAMMALS 审中-公开
标题翻译： RNAi介导的抑制基因表达在哺乳动物中的方法和组合
公开(公告)号：WO2003010180A1
公开(公告)日：2003-02-06
申请号：PCT/US2002/022869
申请日：2002-07-19
申请人： THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY
发明人： KAY, Mark , MCCAFFREY, Anton
IPC分类号： C07H21/02
CPC分类号： A61K31/7105 , A01K67/0275 , A01K2217/075 , A61K31/70 , A61K31/713 , A61K45/06 , A61K48/00 , A61K49/0008
摘要： Methods and compositions are provided for modulating, e.g., reducing, coding sequence expression in mammals. In the subject methods, an effective amount of an RNAi agent, e.g., an interfering ribonucleic acid (such as an siRNA or shRNA) or a transcription template thereof, e.g., a DNA encoding an shRNA, is administered to a non-embryonic mammal, e.g., via a hydrodynamic administration protocol. Also provided are RNAi agent pharmaceutical preparations for use in the subject methods. The subject methods and compositions find use in a variety of different applications, including academic and therapeutic applications.
摘要翻译：提供了用于调节，例如降低哺乳动物序列表达的方法和组合物。在本发明的方法中，向非胚胎哺乳动物施用有效量的RNAi剂，例如干扰性核糖核酸（例如siRNA或shRNA）或其转录模板，例如编码shRNA的DNA，例如，通过流体动力学管理方案。还提供了用于本发明方法的RNAi剂药物制剂。主题方法和组合物可用于各种不同的应用，包括学术和治疗应用。

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