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    Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
    1.
    发明授权
    Methods and compositions for the specific inhibition of gene expression by double-stranded RNA 有权

    公开(公告)号：US10106792B2

    公开(公告)日：2018-10-23

    申请号：US15375730

    申请日：2016-12-12

    申请人： CITY OF HOPE ,  INTEGRATED DNA TECHNOLOGIES, INC.

    发明人： John J. Rossi ,  Mark A. Behlke ,  Dongho Kim

    IPC分类号： C07H21/04 ,  C12N15/113 ,  C12N15/11

    摘要： The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.

    Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
    4.
    发明授权
    Methods and compositions for the specific inhibition of gene expression by double-stranded RNA 有权
    通过双链RNA特异性抑制基因表达的方法和组合物

    公开(公告)号：US09441227B2

    公开(公告)日：2016-09-13

    申请号：US14537578

    申请日：2014-11-10

    申请人： CITY OF HOPE ,  INTEGRATED DNA TECHNOLOGIES, INC.

    发明人： John J. Rossi ,  Mark A. Behlke ,  Dongho Kim

    IPC分类号： C07H21/04 ,  C12N15/113 ,  C12N15/11

    CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/33 ,  C12N2310/53 ,  C12N2310/533 ,  C12N2320/35 ,  C12N2320/51

    摘要： The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.

    摘要翻译： 本发明涉及用于选择性地降低来自细胞中所需靶基因的基因产物的表达以及用于治疗由基因表达引起的疾病的组合物和方法。 更具体地，本发明涉及含有双链RNA（“dsRNA”）的组合物及其制备方法，其能够降低真核细胞中靶基因的表达。 dsRNA具有长度为25至约30个核苷酸的第一寡核苷酸序列和在生物条件下与第一序列退火的第二寡核苷酸序列。 此外，具有至少19个核苷酸的序列长度的dsRNA的序列之一的区域与由靶基因产生的RNA的核苷酸序列充分互补，以通过RNAi机制触发靶RNA的破坏。

    Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
    6.
    发明授权
    Methods and compositions for the specific inhibition of gene expression by double-stranded RNA 有权

    公开(公告)号：US10233450B2

    公开(公告)日：2019-03-19

    申请号：US15849887

    申请日：2017-12-21

    申请人： CITY OF HOPE ,  INTEGRATED DNA TECHNOLOGIES, INC.

    发明人： John J. Rossi ,  Mark A. Behlke ,  Dongho Kim

    IPC分类号： C07H21/04 ,  C12N15/113 ,  C12N15/11

    摘要： The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.

    Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
    8.
    发明授权
    Methods and compositions for the specific inhibition of gene expression by double-stranded RNA 有权
    通过双链RNA特异性抑制基因表达的方法和组合物

    公开(公告)号：US09365849B2

    公开(公告)日：2016-06-14

    申请号：US14019911

    申请日：2013-09-06

    申请人： CITY OF HOPE ,  INTEGRATED DNA TECHNOLOGIES, INC.

    发明人： John J. Rossi ,  Mark A. Behlke ,  Dongho Kim

    IPC分类号： C07H21/04 ,  C12N15/113 ,  C12N15/11

    CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/33 ,  C12N2310/50 ,  C12N2310/51 ,  C12N2320/30 ,  C12N2320/50 ,  C12N2320/51 ,  C12N2330/30

    摘要： The invention provides compositions and methods for selectively reducing the expression of a gene product from a desired target gene, as well as treating diseases caused by expression of the gene. The method involves introducing into the environment of a cell an amount of a double-stranded RNA (dsRNA) such that a sufficient portion of the dsRNA can enter the cytoplasm of the cell to cause a reduction in the expression of the target gene. The dsRNA has a first oligonucleotide sequence that is between 26 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of from about 19 to about 23 nucleotides is complementary to a nucleotide sequence of the RNA produced from the target gene.

    摘要翻译： 本发明提供了选择性地降低来自所需靶基因的基因产物表达以及治疗由基因表达引起的疾病的组合物和方法。 该方法包括向细胞的环境中引入一定量的双链RNA（dsRNA），使得足够部分的dsRNA可以进入细胞的细胞质以引起靶基因表达的降低。 dsRNA具有长度为26至约30个核苷酸的第一寡核苷酸序列和在生物条件下与第一序列退火的第二寡核苷酸序列。 此外，具有约19至约23个核苷酸的序列长度的dsRNA序列之一的区域与由靶基因产生的RNA的核苷酸序列互补。

    METHODS FOR ENHANCING NUCLEIC ACID HYBRIDIZATION
    9.
    发明申请
    METHODS FOR ENHANCING NUCLEIC ACID HYBRIDIZATION 有权
    增加核酸杂交的方法

    公开(公告)号：US20150141635A1

    公开(公告)日：2015-05-21

    申请号：US14578399

    申请日：2014-12-20

    申请人： Integrated DNA Technologies, Inc.

    发明人： Scott Rose ,  Mark A. Behlke ,  Richard Owczarzy ,  Joseph A. Walder ,  Derek M. Thomas ,  Michael R. Marvin

    IPC分类号： C12Q1/68

    CPC分类号： C12Q1/6832 ,  C07H21/04 ,  C12Q1/6818 ,  C12Q1/6851 ,  G01N33/5308 ,  G01N33/542 ,  C12Q2525/185 ,  C12Q2525/197 ,  C12Q2565/101

    摘要： A composition comprising an oligonucleotide having the structure 5′-Y1-L1-X-L2-Y2-3′. Y1 comprises a sequence of one or more DNA or RNA nucleotides, including a first nucleotide N1 having a 3′ phosphate covalently linked to L1. Y2 comprises a sequence of one or more DNA or RNA nucleotides, including a second nucleotide N2 having a 5′ phosphate covalently linked to L2. L1 and L2 each independently are a direct bond or a C1-C7 alkyl, alkynyl, alkenyl, heteroalkyl, substituted alkyl, aryl, heteroaryl, substituted aryl, cycloalkyl, alkylaryl, or alkoxyl group. X is R1 is a hydrogen or a C1-C8 alkyl. M is a label or ligand comprising a fused polycyclic aromatic moiety.

    摘要翻译： 包含具有结构5'-Y1-L1-X-L2-Y2-3'的寡核苷酸的组合物。 Y1包含一个或多个DNA或RNA核苷酸的序列，包括与L1共价连接的3'磷酸的第一核苷酸N1。 Y2包含一个或多个DNA或RNA核苷酸的序列，包括与L2共价连接的5'磷酸二酯的第二核苷酸N2。 L 1和L 2各自独立地为直接键或C 1 -C 7烷基，炔基，烯基，杂烷基，取代的烷基，芳基，杂芳基，取代的芳基，环烷基，烷基芳基或烷氧基。 X是R 1是氢或C 1 -C 8烷基。 M是包含稠合多环芳族部分的标记或配体。