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    Methods and Compositions for Regulated Expressions of Nucleic Acid at Post-Transcriptional Level
    1.
    发明申请
    Methods and Compositions for Regulated Expressions of Nucleic Acid at Post-Transcriptional Level 审中-公开
    转录水平后核酸调节表达的方法和组成

    公开(公告)号：US20100196335A1

    公开(公告)日：2010-08-05

    申请号：US11919267

    申请日：2006-04-28

    申请人： Richard J. Samulski ,  Kyson Xiaohuai Chou

    发明人： Richard J. Samulski ,  Kyson Xiaohuai Chou

    IPC分类号： A61K35/12 ,  C07H21/02 ,  C12N15/74 ,  C12N5/10 ,  A61K31/7088 ,  C12P21/00 ,  C12P19/34 ,  A61K31/7105 ,  C12Q1/68 ,  A61P43/00

    CPC分类号： C12N15/85 ,  C12N15/111 ,  C12N15/67 ,  C12N15/8509 ,  C12N2310/11 ,  C12N2320/33 ,  C12N2840/44 ,  C12N2840/445

    摘要： The present invention provides an isolated nucleic acid comprising: a) at least one first nucleotide sequence encoding a heterologous nucleotide sequence of interest; and b) at least two second heterologous nucleotide sequences, wherein each second heterologous nucleotide sequences comprises: i) a first set of splice elements defining a first intron that is removed by splicing to produce a first RNA molecule that imparts a biological function in the absence of activity at a second set of splice elements; and ii) the second set of splice elements defining one or more introns different from said first intron, wherein said one or more introns different from said first intron are removed by splicing to produce no RNA molecule and/or a second RNA molecule that docs not impart a biological function, when said second set of splice elements is active. Further provided are methods of using the nucleic acid of this invention to regulate transgene expression.

    摘要翻译： 本发明提供了一种分离的核酸，其包含：a）至少一个编码感兴趣的异源核苷酸序列的第一核苷酸序列; 和b）至少两个第二异源核苷酸序列，其中每个第二异源核苷酸序列包括：i）限定第一内含子的第一组剪接元件，其通过剪接除去以产生在不存在时赋予生物学功能的第一RNA分子 在第二组拼接元件上的活动; 和ii）限定与所述第一内含子不同的一个或多个内含子的第二组拼接元件，其中与所述第一内含子不同的所述一个或多个内含子通过剪接除去以产生不分泌RNA的RNA分子和/或第二RNA分子 当所述第二组接头元件是活动的时，赋予生物功能。 还提供了使用本发明的核酸调节转基因表达的方法。

    AAV TRANSDUCTION OF MUSCLE TISSUE
    4.
    发明申请
    AAV TRANSDUCTION OF MUSCLE TISSUE 审中-公开
    肌肉组织的AAV转导

    公开(公告)号：US20090298922A1

    公开(公告)日：2009-12-03

    申请号：US12537806

    申请日：2009-08-07

    申请人： Xiao Xiao ,  Richard J. Samulski ,  Juan Li

    发明人： Xiao Xiao ,  Richard J. Samulski ,  Juan Li

    IPC分类号： A61K48/00

    CPC分类号： C12N15/86 ,  A61K48/00 ,  C12N2750/14143

    摘要： A method of expressing a gene product in the muscle tissue of an animal, which comprises administering a recombinant AAV vector to the muscle tissue of the animal, wherein the vector comprises a non-AAV gene of interest ligated into an AAV vector genome.

    摘要翻译： 一种在动物的肌肉组织中表达基因产物的方法，其包括向所述动物的肌肉组织施用重组AAV载体，其中所述载体包含连接到AAV载体基因组中的目的非AAV基因。

    AAV transduction vectors
    6.
    发明授权
    AAV transduction vectors 失效
    AAV转导载体

    公开(公告)号：US5139941A

    公开(公告)日：1992-08-18

    申请号：US785224

    申请日：1991-10-25

    申请人： Nicholas Muzyczka ,  Paul L. Hermonat ,  Kenneth I. Berns ,  Richard J. Samulski

    发明人： Nicholas Muzyczka ,  Paul L. Hermonat ,  Kenneth I. Berns ,  Richard J. Samulski

    IPC分类号： C12N15/864

    CPC分类号： C12N15/86 ,  C12N2750/14143

    摘要： A hybrid gene vector suitable for introducing foreign DNA into a mammalian cell comprising the foreign DNA ligated to an AAV genome; a method of constructing the hybrid gene vector; a method of transducing foreign DNA into mammalian cells comprising infecting the cells with the above hybrid gene vector and a method of rescuing foreign DNA from mammalian cells utilizing helper virus.

    摘要翻译： 一种适用于将外源DNA引入哺乳动物细胞的杂交基因载体，其包含与AAV基因组连接的外源DNA; 构建杂交基因载体的方法; 将外源DNA转导入哺乳动物细胞的方法，包括用上述杂交基因载体感染细胞，以及利用辅助病毒从哺乳动物细胞中拯救外来DNA的方法。

    Helper-free stocks of recombinant adeno-associated virus vectors
    9.
    发明授权
    Helper-free stocks of recombinant adeno-associated virus vectors 失效

    公开(公告)号：US5436146A

    公开(公告)日：1995-07-25

    申请号：US7399

    申请日：1993-01-21

    申请人： Thomas E. Shenk ,  Richard J. Samulski ,  Long-Sheng Chang

    发明人： Thomas E. Shenk ,  Richard J. Samulski ,  Long-Sheng Chang

    IPC分类号： C12N5/02 ,  C12N7/01 ,  C12N15/00 ,  C12N15/09 ,  C12N15/63 ,  C12N15/70 ,  C12N15/74 ,  C12N15/86 ,  C12N15/864 ,  C12Q1/68 ,  C12Q1/70 ,  G01N33/53 ,  C12N5/10 ,  C12N15/35

    CPC分类号： C12N15/86 ,  C12N2750/14143

    摘要： The present invention relates to a method for producing helper-free stocks of recombinant adeno-associated virus (rAAV) which can be used to efficiently and stably transduce foreign genes into host cells or organisms. The method comprises the cotransfection of eukaryotic cells with rAAV and with helper AAV DNA in the presence of helper virus (e.g. adenovirus or herpesvirus) such that the helper AAV DNA is not associated with virion formation. The crux of the invention lies in the inability of the helper AAV DNA to recombine with rAAV vector, thereby preventing the generation of wild-type virus. In a specific embodiment of the invention, the vector comprises a recombinant AAV genome containing only the terminal regions of the AAV chromosome bracketing a non-viral gene, and the helper AAV DNA comprises a recombinant AAV genome containing that part of the AAV genome which is not present in the vector, and in which the AAV terminal regions are replaced by adenovirus sequences. In a further embodiment of the invention, cell lines are created which incorporate helper AAV DNA which can directly produce substantially pure recombinant AAV virus. The pure stocks of recombinant AAV produced according to the invention provide an AAV viral expression vector system with increased yield of recombinant virus, improved efficiency, higher definition, and greater safety than presently used systems.