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    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：US08809515B2

    公开(公告)日：2014-08-19

    申请号：US13178806

    申请日：2011-07-08

    申请人： John J. Rossi ,  Mark A. Behlke ,  Dongho Kim

    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 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。

    Nucleolar targeting of therapeutics against HIV
    5.
    发明授权
    Nucleolar targeting of therapeutics against HIV 有权
    针对艾滋病毒治疗药物的核仁靶点

    公开(公告)号：US08227442B2

    公开(公告)日：2012-07-24

    申请号：US12835333

    申请日：2010-07-13

    申请人： John J. Rossi ,  Alessandro Michienzi

    发明人： John J. Rossi ,  Alessandro Michienzi

    IPC分类号： C12N15/11 ,  C07H21/04

    CPC分类号： C12N15/1132 ,  C12N2310/121 ,  C12N2310/3513

    摘要： The HIV regulatory proteins Tat and Rev accumulate in nucleoli of human cells. No functional role has been attributed to this localization. Recently it was demonstrated that expression of Rev induces nucleolar re-localization of some nuclear factors involved in Rev export. Thus, it is likely that the nucleolus plays a critical role in Rev-mediated export of singly spliced and unspliced HIV-1 RNAs. As a test for trafficking of HIV-1 RNAs into the nucleolus, a hammerhead ribozyme which specifically cleaves HIV-1 RNA was joined to the U16 snoRNA resulting in accumulation of the ribozyme within nucleoli of human cells. Stably transduced human T-cells expressing this nucleolar localized ribozyme dramatically suppressed HIV-1 replication, confirming a possible trafficking of the HIV RNA through the nucleoli of human cells. In addition, a TAR element which binds Tat was joined to the U16 snoRNA, also resulting in localization in the nucleoli and inhibiting HIV replication.

    摘要翻译： HIV调节蛋白Tat和Rev在人细胞的核仁中积累。 这个本地化没有任何功能的作用。 最近有证据表明，Rev的表达诱导了涉及Rev出口的一些核因子的核仁重新定位。 因此，核仁可能在Rev-mediated转录单个拼接和未被剪接的HIV-1 RNAs的出口中起关键作用。 作为将HIV-1 RNA转运入核仁的测试，将特异性切割HIV-1 RNA的锤头核酶与U16 snoRNA连接，导致核酶在人细胞核内的积累。 稳定转导的表达这种核仁定位核酶的人类T细胞显着抑制HIV-1复制，证实可能通过人类细胞的核仁运送HIV RNA。 此外，结合Tat的TAR元件连接到U16 snoRNA，也导致在核仁中的定位并抑制HIV复制。

    MICRO RNAS AND THEIR METHODS OF USE FOR THE TREATMENT AND DIAGNOSIS OF SCHIZOPHRENIA AND SCHIZOPHRENIA SPECTRUM DISORDERS
    9.
    发明申请
    MICRO RNAS AND THEIR METHODS OF USE FOR THE TREATMENT AND DIAGNOSIS OF SCHIZOPHRENIA AND SCHIZOPHRENIA SPECTRUM DISORDERS 有权
    MICRO RNAS及其用于治疗和诊断SCHIZOPHRENIA和SCHIZOPHRENIA光谱病症的方法

    公开(公告)号：US20100009367A1

    公开(公告)日：2010-01-14

    申请号：US12484145

    申请日：2009-06-12

    申请人： Steve S. Sommer ,  John J. Rossi

    发明人： Steve S. Sommer ,  John J. Rossi

    IPC分类号： C12Q1/68

    CPC分类号： C12Q1/6883 ,  C12Q2600/156 ,  C12Q2600/158 ,  C12Q2600/178

    摘要： A method of diagnosing, assessing susceptibility, and/or treating schizophrenia involving the identification and/or observation of microRNAs (miRNA) and variant miRNA are provided. Micro RNAs alleles associated with schizophrenia and schizophrenia spectrum disorders were identified and ultra-rare variants in the precursor or mature miRNA were identified. Functional analyses of ectopically expressed copies of the variant miRNA precursors demonstrate loss of function, gain of function and altered expression levels. The present invention also provides methods for selecting a preferred therapy for a particular subject or group of subjects or individuals at risk for or suffering from schizophrenia or psychosis by use of miRNAs.

    摘要翻译： 提供了诊断，评估易感性和/或治疗涉及微小RNA（miRNA）和变体miRNA的鉴定和/或观察的精神分裂症的方法。 鉴定了与精神分裂症和精神分裂症谱系疾病相关的微RNA等位基因，并鉴定了前体或成熟miRNA中的超罕见变异体。 对异源miRNA前体的异位表达拷贝的功能分析表明功能丧失，功能增加和表达水平改变。 本发明还提供了通过使用miRNA选择患有或患有精神分裂症或精神病的受试者或个体的特定受试者或个体的优选疗法的方法。