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    SPLICE INHIBITING OLIGONUCLEOTIDES
    3.
    发明申请
    SPLICE INHIBITING OLIGONUCLEOTIDES 审中-公开

    公开(公告)号：US20200216850A1

    公开(公告)日：2020-07-09

    申请号：US16648822

    申请日：2018-09-21

    申请人： CITY OF HOPE

    发明人： John C. Burnett ,  Elizabeth Epps ,  John J. Rossi

    IPC分类号： C12N15/113 ,  C12N15/86

    摘要： MicroRNAs embedded within an intron, which are called ‘mirtrons,’ can be used as a platform for expressing one or more shRNA or miRNA mimics in a lentiviral vector. The inventors developed a strategy to improve lentiviral titering by reducing the production of shRNA/miRNA from the vector during packaging through the introduction of splice-inhibiting antisense oligonucleotides during vector packaging, which inhibit the splicing of the mirtron and subsequent processing of the shRNAs/miRNAs. In an aspect is provided a kit comprising an oligonucleotide comprising a mirtron splice site binding sequence and a lentiviral packaging system. In an aspect is provided a method for producing a lentivirus. The method comprises the step of transfecting a cell with an oligonucleotide comprising a mirtron splice site binding sequence and a lentiviral packaging system; thereby producing the lentivirus.

    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：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.

    RNAI molecules with non-watson crick pairing based on artificial mutation consensus sequences to counter escape mutations
    8.
    发明授权
    RNAI molecules with non-watson crick pairing based on artificial mutation consensus sequences to counter escape mutations 有权
    基于人工突变共有序列的非沃顿克里克配对的RNAI分子来对抗逃逸突变

    公开(公告)号：US09212363B2

    公开(公告)日：2015-12-15

    申请号：US13842977

    申请日：2013-03-15

    申请人： CITY OF HOPE

    发明人： John J. Rossi ,  Ulrike Jung

    IPC分类号： C07H21/04 ,  C12N15/113

    CPC分类号： C12N15/1132 ,  C12N15/1131 ,  C12N2310/533 ,  C12N2320/34 ,  C12N2310/14 ,  C12N2310/531

    摘要： Universal RNA interference (RNAi) molecules having an inhibitory RNA sequence which binds a target pathologic RNA sequence are provided according to some embodiments. Such RNAi molecules bind the target pathologic RNA sequence via at least one non-Watson Crick paired base. In some embodiments, the target pathologic RNA sequence is a target viral RNA sequence derived from a human immunodeficiency HIV virus, a hepatitis B virus (HBV), a hepatitis C virus (HCV), or an influenza virus.

    摘要翻译： 根据一些实施方案提供具有结合目标病理RNA序列的抑制性RNA序列的通用RNA干扰（RNAi）分子。 这样的RNAi分子通过至少一个非沃森克里克配对基团结合目标病理RNA序列。 在一些实施方案中，靶病理RNA序列是衍生自人类免疫缺陷型HIV病毒，乙型肝炎病毒（HBV），丙型肝炎病毒（HCV）或流感病毒的靶病毒RNA序列。