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1.发明授权公开(公告)号：US07691995B2
公开(公告)日：2010-04-06
申请号：US10195034
申请日：2002-07-12
IPC分类号： C07H21/04
CPC分类号： C12N15/113 , A01K2217/05 , A01K2227/105 , A01K2267/03 , C12N15/111 , C12N15/8509 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/30 , C12N2330/30 , C12N2330/51 , C12N2517/02 , C12N2799/021 , C12N2830/008
摘要： The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.
摘要翻译： 本发明提供了工程化的RNA前体，其在细胞中表达时被细胞加工以产生靶向小干扰RNA（siRNA），其使用细胞自身的RNA干扰（RNAi）途径选择性沉默靶基因（通过切割特异性mRNA）。 通过将具有合适的调节序列将这些工程化RNA前体编码的核酸分子引入细胞体内，工程化RNA前体的表达可以在时间和空间上选择性地控制，即在特定时间和/或特别是组织，器官或 细胞。
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2.发明授权公开(公告)号：US08557785B2
公开(公告)日：2013-10-15
申请号：US12729360
申请日：2010-03-23
IPC分类号： C12N15/11
CPC分类号： C12N15/113 , A01K2217/05 , A01K2227/105 , A01K2267/03 , C12N15/111 , C12N15/8509 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/30 , C12N2330/30 , C12N2330/51 , C12N2517/02 , C12N2799/021 , C12N2830/008
摘要： The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.
摘要翻译： 本发明提供了工程化的RNA前体，其在细胞中表达时被细胞加工以产生靶向小干扰RNA（siRNA），其使用细胞自身的RNA干扰（RNAi）途径选择性沉默靶基因（通过切割特异性mRNA）。 通过将具有合适的调节序列将这些工程化RNA前体编码的核酸分子引入细胞体内，工程化RNA前体的表达可以在时间和空间上选择性地控制，即在特定时间和/或特别是组织，器官或 细胞。
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公开(公告)号：US08530438B2
公开(公告)日：2013-09-10
申请号：US12727783
申请日：2010-03-19
IPC分类号： C12N15/11
CPC分类号： C12N15/113 , A01K2217/05 , A01K2227/105 , A01K2267/03 , C12N15/111 , C12N15/8509 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/30 , C12N2330/30 , C12N2330/51 , C12N2517/02 , C12N2799/021 , C12N2830/008
摘要： The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.
摘要翻译： 本发明提供了工程化的RNA前体，其在细胞中表达时被细胞加工以产生靶向小干扰RNA（siRNA），其使用细胞自身的RNA干扰（RNAi）途径选择性沉默靶基因（通过切割特异性mRNA）。 通过将具有合适的调节序列将这些工程化RNA前体编码的核酸分子引入细胞体内，工程化RNA前体的表达可以在时间和空间上选择性地控制，即在特定时间和/或特别是组织，器官或 细胞。
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4.发明授权公开(公告)号：US08232260B2
公开(公告)日：2012-07-31
申请号：US13031522
申请日：2011-02-21
IPC分类号： C12N15/11
CPC分类号： C12N15/113 , A01K2217/05 , A01K2227/105 , A01K2267/03 , C12N15/111 , C12N15/8509 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/30 , C12N2330/30 , C12N2330/51 , C12N2517/02 , C12N2799/021 , C12N2830/008
摘要： The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vitro with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.
摘要翻译： 本发明提供了工程化的RNA前体，其在细胞中表达时被细胞加工以产生靶向小干扰RNA（siRNA），其使用细胞自身的RNA干扰（RNAi）途径选择性沉默靶基因（通过切割特异性mRNA）。 通过将具有合适调控序列的这些工程化RNA前体编码的核酸分子引入体外，工程改造的RNA前体的表达可以在时间上和空间上选择性地控制，即在特定时间和/或特别是组织，器官或 细胞。
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5.发明申请公开(公告)号：US20080200420A1
公开(公告)日：2008-08-21
申请号：US12079531
申请日：2008-03-26
CPC分类号： C12N15/113 , A01K2217/05 , A01K2227/105 , A01K2267/03 , C12N15/111 , C12N15/8509 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/30 , C12N2330/30 , C12N2330/51 , C12N2517/02 , C12N2799/021 , C12N2830/008
摘要： The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.
摘要翻译： 本发明提供了工程化的RNA前体，其在细胞中表达时被细胞加工以产生靶向小干扰RNA（siRNA），其使用细胞自身的RNA干扰（RNAi）途径选择性沉默靶基因（通过切割特异性mRNA）。 通过将具有合适的调节序列将这些工程化RNA前体编码的核酸分子引入细胞体内，工程化RNA前体的表达可以在时间和空间上选择性地控制，即在特定时间和/或特别是组织，器官或 细胞。
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6.发明申请公开(公告)号：US20060009402A1
公开(公告)日：2006-01-12
申请号：US10195034
申请日：2002-07-12
IPC分类号： A61K48/00
CPC分类号： C12N15/113 , A01K2217/05 , A01K2227/105 , A01K2267/03 , C12N15/111 , C12N15/8509 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/30 , C12N2330/30 , C12N2330/51 , C12N2517/02 , C12N2799/021 , C12N2830/008
摘要： The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.
摘要翻译： 本发明提供了工程化的RNA前体，其在细胞中表达时被细胞加工以产生靶向小干扰RNA（siRNA），其使用细胞自身的RNA干扰（RNAi）途径选择性沉默靶基因（通过切割特异性mRNA）。 通过将具有合适的调节序列将这些工程化RNA前体编码的核酸分子引入细胞体内，工程化RNA前体的表达可以在时间和空间上选择性地控制，即在特定时间和/或特别是组织，器官或 细胞。
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公开(公告)号：US08598143B2
公开(公告)日：2013-12-03
申请号：US12273673
申请日：2008-11-19
IPC分类号： A01N43/04
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/321 , C12N2320/50 , C12Q1/6897 , C12N2310/3521
摘要： The present invention relates to the discovery of a method for inhibiting RNA silencing in a target sequence-specific manner. RNA silencing requires a set of conserved cellular factors to suppress expression of gene-encoded polypeptide. The invention provides compositions for sequence-specific inactivation of the RISC component of the RNA silencing pathway, and methods of use thereof. The RISC inactivators of the present invention enable a variety of methods for identifying and characterizing miRNAs and siRNAs, RISC-associated factors, and agents capable of modulating RNA silencing. Therapeutic methods and compositions incorporating RISC inactivators and therapeutic agents identified through use of RISC inactivators are also featured.
摘要翻译： 本发明涉及以目标序列特异性方式发现抑制RNA沉默的方法。 RNA沉默需要一组保守的细胞因子来抑制基因编码的多肽的表达。 本发明提供了用于RNA沉默途径的RISC组分的序列特异性失活的组合物及其使用方法。 本发明的RISC灭活剂能够用于鉴定和表征miRNA和siRNA，RISC相关因子和能调节RNA沉默的多种方法。 还列出了通过使用RISC灭活剂鉴定的包含RISC灭活剂和治疗剂的治疗方法和组合物。
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8.发明授权公开(公告)号：US08309704B2
公开(公告)日：2012-11-13
申请号：US10859321
申请日：2004-06-02
CPC分类号： C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/331 , C12N2310/53 , C12N2320/11 , C12N2320/50
摘要： The present invention provides methods of enhancing the efficacy and specificity of RNAi. The invention also provides compositions for mediating RNAi. In particular, the invention provides siRNAs, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.
摘要翻译： 本发明提供增强RNAi功效和特异性的方法。 本发明还提供用于介导RNAi的组合物。 特别地，本发明提供具有改善的靶基因沉默的特异性和功效的siRNA，shRNA，载体和转基因。 还介绍了治疗方法。
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9.发明授权Methods and compositions for enhancing the efficacy and specificity of RNA silencing 有权用于增强RNA沉默的功效和特异性的方法和组合物公开(公告)号：US08304530B2
公开(公告)日：2012-11-06
申请号：US12729892
申请日：2010-03-23
CPC分类号： C12N15/11 , A61K38/00 , A61K48/00 , C12N15/111 , C12N15/113 , C12N2310/111 , C12N2310/14 , C12N2310/331 , C12N2310/333 , C12N2310/336 , C12N2320/51
摘要： The present invention provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.
摘要翻译： 本发明提供增强RNA沉默的功效和特异性的方法。 本发明还提供介导RNA沉默的组合物。 特别地，本发明提供了siRNA，siRNA样分子，shRNA，载体和转基因，其具有改善的靶基因沉默的特异性和功效。 还介绍了治疗方法。
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公开(公告)号：US08309705B2
公开(公告)日：2012-11-13
申请号：US12748937
申请日：2010-03-29
CPC分类号： C12N15/11 , A61K38/00 , A61K48/00 , C12N15/111 , C12N15/113 , C12N2310/111 , C12N2310/14 , C12N2310/331 , C12N2310/333 , C12N2310/336 , C12N2320/51
摘要： The present invention provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.
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