Abstract: Provided herein are compositions comprising oligomeric compounds. In certain embodiments, the oligomeric compounds are useful as miRNA mimics. The oligomeric compounds may mimic the activity of miR-34. Also provided herein are methods for the treatment of cancer.

Abstract: A method is disclosed for reducing the level of gossypol in cottonseed. The method generally includes selectively inducing RNA gene silencing in the seed of a transgenic cotton plant, to interfere with expression of the ?-cadinene synthase gene or the ?-cadinene-8-hydroxylase gene in the seed of the cotton plant without substantially affecting expression of that gene in the foliage, floral parts, and roots of the plant. The transgenic cotton plant comprises at least one of a ?-cadinene synthase gene trigger sequence and/or a ?-cadinene-8-hydroxylase gene trigger sequence operably linked to one or more a seed-specific promoter gene sequences, and the trigger sequence(s) is/are able to induce RNA gene silencing when expressed in cottonseed of the plant.

Abstract: The present invention relates to the discovery of (SNPs) significantly associated with Huntington's disease (HD). The present invention utilizes RNA silencing technology (e.g. RNAi) against such SNPs optimally combined with select additional SNP targeting silencing agents, thereby resulting in an effective treatment of significantly-sized patient populations. Silencing agents having enhanced discriminatory properties are also featured.

Abstract: The present invention provides agents, compositions, constructs and methods for silencing HCV polynucleotides, as well as methods and compositions for treating or preventing HCV infection in a mammalian cell. In one aspect, the present invention provides an agent or composition comprising at least one double-stranded RNA effector molecule or complex. The double-stranded RNA effector molecule or complex comprises: (1) a sequence of at least 19 nucleotides having at least 90% identity with a nucleotide sequence within HCV Conserved Region 1 (SEQ ID NO: 2), HCV Conserved Region 2 (SEQ ID NO: 3), HCV Conserved Region 5 (SEQ ID NO: 4), (ATR)-1 (SEQ ID NO: 86), ATR-2 (SEQ ID NO: 87), ATR-3 (SEQ ID NO: 88), ATR-4 (SEQ ID NO: 89); and (2) its complementary sequence. In another aspect, the present invention provides a construct suitable for replication in a host cell, and/or suitable for expression of an RNA molecule or complex of the invention in vitro or in vivo.

Abstract: A novel network of tumorigenic prognostic factors is identified that plays a critical role in advanced pancreatic cancer (PC) pathogenesis. This interactome is interconnected through a central tumor suppressive microRNA, miR-198, which is able to both directly and indirectly modulate expression of the various members of this network to alter the molecular makeup of pancreatic tumors. When this tumor signature network is intact, miR-198 expression is reduced and patient survival is dismal; patients with higher miR-198 present an altered tumor signature network, better prognosis and increased survival. Further, MiR-198 replacement reverses tumorigenicity in vitro and in vivo. embodiment of the disclosure is a method of treating cancer in an individual, comprising the step of increasing the level of active microRNA-198 molecules in the pancreatic cancer tumor cells of the individual by an amount sufficient to cause an improvement in the pancreatic cancer in the individual.

Abstract: The present invention provides oligomeric compounds and uses thereof. In certain embodiments, such oligomeric compounds are useful as antisense compounds. Certain such antisense compounds are useful as RNase H antisense compounds or as RNAi compounds.

Abstract: The invention includes a method of treating an intraocular disorder in a mammal, the method comprising administering to the mammal a Very Late Antigen-4 (VLA-4) antagonist for the treatment of selected ocular disorders.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of NANOG, in particular, by targeting natural antisense polynucleotides of NANOG. The invention also related to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of NANOG.

Abstract: The present invention provides isolated polynucleotide sequences encoding ?-D-N-acetylhexosaminidase. The present invention further provides DNA construct comprising the polynucleotide sequence coding for ?-D-N-acetylhexosaminidase in sense or antisense orientation, RNAi construct, recombinant vector comprising the construct and host cells comprising the recombinant vector disclosed in the present invention. The present invention further provides transgenic plant, plant cell, transgenic progeny and seeds expressing the polynucleotide with reduced ?-D-N-acetylhexosaminidase protein accumulation, having enhanced fruit shelf life.

Abstract: A method for treating cells and/or nuclear transfer units and/or stem cells in culture with such compounds, individually or in combinations, is described. The method results in a globally hypomethylated genome and a restoration of cell differentiation and/or developmental potential, or potentiality. In addition, a method for the in vitro production of reprogrammed cells which have had differentiation potential (totipotential, pluripotential, or multipotential) restored by demethylating the genome is described.

Abstract: Synthesis and uses of conformationally restricted nucleomonomers (CRN) to prepare nucleic acid compounds. Methods for preparing nucleomonomers for nucleic acid compounds in high yields and in multi-gram scale for therapeutic modalities useful for treating or preventing diseases or disorders by up- or down-regulating the expression of genes and other nucleic acid based regulatory systems in a cell.

Abstract: Provided herein are compositions and methods for the modulation of miR-214 for the treatment and/or prevention of fibrosis and fibroproliferative conditions.

Abstract: The present invention relate to antisense oligonucleotides that modulate the expression of and/or function of Transcription factor E3 (TFE3) and/or Insulin Receptor Substrate 2 (IRS2) polynucleotides, in particular, by targeting natural antisense polynucleotides of Transcription factor E3 (TFE3) and/or Insulin Receptor Substrate 2 (IRS2). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of TFE3 and/or IRS2.

Abstract: The present invention relates to the field of RNA-mediated gene silencing in insect species. The present invention is based, in part, on the inventors' sequencing of genes from eucalyptus invasive species Bronze bug pest, Thaumastocoris peregrinus. In certain aspects, the invention provides Bronze bug nucleic acids, derivatives thereof and the use of such nucleic acids and derivatives as Bronze bug control agents.

Abstract: The present invention relates to; a pharmaceutical compostion capable of enhancing immunity against viruses by specifically decreasing the expression of the OASL1 protein; and a method for screening for a material capable of being used as an antiviral agent by comparing the amount of expression of the OASL1 protein.

Abstract: This invention relates to the field of molecular physiology. Specifically, this invention relates to the prevention and/or treatment of acute inflammation of the respiratory tract, especially acute lung injury (ALI) or acute respiratory distress syndrome (ARDS). Levels of CCL7 have been demonstrated to be increased in patients suffering from such conditions and animal models of such conditions. Antagonists of CCL7 and/or other members of the PAR1-CCL7 axis, or CCL2 can be used to prevent and/or treat these conditions.

Abstract: The present invention relates to control of plant pathogens, particularly fungi or oomycetes, by inhibiting one or more biological functions, particularly by inhibiting saccharopine dehydrogenase gene(s) using RNA interference. The invention provides methods and compositions using RNA interference of plant pathogens target genes for such control. The invention is also directed to methods for making transgenic plants tolerant to said plant pathogens, and to transgenic plants and seeds generated thereof.

Abstract: The invention relates to oligonucleotides for inducing skipping of exon 55 of the dystrophin gene. The invention also relates to methods of inducing exon 55 skipping using the oligonucleotides.

Abstract: The present invention provides nucleosides and oligonucleotides comprising a 5? phosphate mimics of formula (IVc) or (Vc), One aspect of the present invention relates to modified nucleosides and oligonucleotides comprising such dinucleotide of formula (Ia). Another aspect of the invention relates to a method of inhibiting the expression of a gene in call, the method comprising (a) contacting an oligonucleotide of the invention with the cell; and (b) maintaining the cell from step (a) for a time sufficient to obtain degradation of the mRNA of the target gene.

Abstract: The present invention relates to the field of RNA-mediated gene silencing in insect species. The present invention is based, in part, on the inventors' sequencing of genes from eucalyptus invasive species Gb pest, Glycaspis brimblecombei. In certain aspects, the invention provides Gb nucleic acids, derivatives thereof and the use of such nucleic acids and derivatives as Gb control agents.

Abstract: The invention relates to methods for the prophylaxis and treatment of breast cancer using one or more antagonists of artemin function, such as anti-artemin polynucleotides or anti-artemin antibodies and antibody fragments, and uses of these antagonists. In particular, the invention relates to the resensitisation of therapy-resistance breast cancer cells to anti-cancer therapies by antagonism of artemin functionality.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Colony-stimulating factor 3 (CSF3), in particular, by targeting natural antisense polynucleotides of Colony-stimulating factor 3 (CSF3). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of CSF3.

Abstract: Compositions and methods for inhibiting the actions of non-coding RNAs such as miRNAs and piRNAs are provided. The compositions comprise single or double stranded oligonucleotides conjugated with Minor Groove Binders (“MGBs”). The oligonucleotides can vary in length, can contain nucleotides having one or more modifications, and have regions that are substantially complementary to one or more mature miRNAs or piRNAs.

Abstract: The present invention provides methods to reduce or inhibit weight gain by administering inhibitors of the SirT1 protein to a subject. Methods to identify such inhibitors are also disclosed.

Abstract: The present invention relates to a technology of enhancing sensitivity to radiotherapy using microRNA, more particularly to a radiosensitizer composition comprising at least one selected from the group consisting of microRNA-26b, microRNA-203 and microRNA-200c, an anticancer supplement, and a method for enhancing sensitivity to radiotherapy of cancer cells using the same.

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.

Abstract: An object of the present invention is to provide a novel double-stranded RNA that has high nuclease resistance and high cellular uptake efficiency, and that is capable of producing an excellent RNA interference effect. The present invention provides a lipid-modified double-stranded RNA comprising a sense strand having a nucleotide sequence complementary to a target sequence, and an antisense strand having a nucleotide sequence complementary to the sense strand, the double-stranded RNA being capable of inhibiting the expression of the target gene, the sense strand having a lipid linked to at least one of the first to sixth nucleotides from the 5? end side directly or via a linker.

Abstract: Methods and compositions useful in target sequence suppression and target sequence validation are described. Polynucleotide constructs useful for gene silencing, as well as cells, plants and seeds comprising the polynucleotides and a method for using microRNAs to silence a target sequence are also described.

Abstract: An agent for inhibiting translesion DNA replication comprises a non-natural adenine ribose analog represented by those as set forth in FIG. 1.

Abstract: The present invention relates to methods for diagnosing, staging, prognosticating and treating melanoma based on evaluating the expression of specific patterns of oncogenic or suppressive microRNA (miR) molecules in a patient in need thereof.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Discs large homolog (DLG), in particular, by targeting natural antisense polynucleotides of Discs large homolog (DLG). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of DLG.

Abstract: The present invention provides an aptamer that binds to prostate-specific antigen (PSA), the aptamer including: a preceding region having a random polynucleotide sequence consisting of from 1 to 10 nucleotides; a first region, at a 3? end of the preceding region, consisting of nnnnCT wherein each n is independently selected from A, T, G and C; a second region consisting of nnCTTT wherein each n is independently selected from A, T, G and C, and at least one part of the second region is complementary to the first region; and a third region positioned between the first region and the second region and consisting of a random polynucleotide sequence having from 3 to 30 nucleotides.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Methionine Sulfoxide Reductase A (MSRA), in particular, by targeting natural antisense polynucleotides of Methionine Sulfoxide Reductase A (MSRA). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of MSRA.

Abstract: The invention inter alia relates to new pharmaceutical compositions comprising miRNAs miR-130a, miR-203 and miR-205, and their use for the treatment of cancer, in particular prostate cancer.

Abstract: The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing an isolate muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method.

Abstract: The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent.

Abstract: Functionalized fluorine containing phthalocyanine molecules, methods of making, and methods of use in diagnostic applications and disease treatment are disclosed herein. In some embodiments, the fluorine containing phthalocyanine molecules are functionalized with a reactive functional group or at least one cancer-targeting ligand (CTL). The CTL can facilitate more efficient binding and/or internalization to a cancer cell than to a healthy cell. The CTL can inhibit expression of oncoprotein in some embodiments. The pthalocyanine moiety can be used in diagnostic applications, such as fluorescence labeling of a cancer cell, and/or treatment applications, such as catalyzing formation of a reactive oxygen species (ROS) which can contribute to cell death of a cancer cell.

Abstract: The invention provides a microRNA inhibitor that has two or more sequences complementary to the sequence of microRNA to be the target of inhibition, which two or more complementary sequences are linked via one or more linker residues.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Down Syndrome Gene, in particular, by targeting natural antisense polynucleotides of a Down Syndrome Gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Down Syndrome Genes.

Abstract: The present invention includes methods and biomarkers for diagnosing or detecting colorectal cancer metastasis in a human subject by comparing the Alu repeat methylation level in the biological sample to an Alu repeat methylation control level from a normal non-cancerous sample from the human subject, wherein a decrease in the Alu repeat methylation level is indicative of colorectal cancer and colorectal cancer metastasis.

Abstract: The invention relates to a method of intracellularly controlling amounts of gene products, which can increase an amount of gene product intracellularly, comprising a step of introducing into the cell a substance having a sequence complementary to the base sequence of mRNA corresponding to the gene product, its precursor or another substance which can have equivalent action in the cell.

Abstract: Treatment of fibrosis and fibrotic diseases, disorders, and conditions, and associated methods, compositions, formulations and articles.

Abstract: The present invention provides a method for inhibiting cell growth, a nucleic acid molecule useful as an anticancer agent, and a method for screening novel anticancer agents. In the present invention, inhibitory effects on expression of NEK10 variant gene or inhibitory effects on activity of NEK10 variant protein are obtained in cells by transfecting cells with a nucleic acid molecule having an RNA interference effect on NEK10 variant gene. The present invention also provides a method for screening anticancer agents by using this inhibitory effect as an indicator.

Abstract: This invention relates to compounds, compositions, and methods useful for reducing MCL1 target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Abstract: The present invention relates to agents, compositions and methods for inhibiting the expression of a target gene, comprising an RNAi agent bearing at least one galactosyl moiety. These are useful for delivering the gene expression inhibiting activity to cells, particularly hepatocytes, and more particularly in therapeutic applications.

Abstract: The present invention relates to methods for controlling pest infestation using double stranded RNA molecules. The invention provides methods for producing transgenic cells expressing the double stranded RNA molecules, as well as compositions and commodity products containing or treated with such molecules.

Abstract: The present invention relates to epilepsy-inducing brain somatic mutations which are associated with intractable epilepsy caused by malformations of cortical development, and uses thereof. More particularly, the present invention relates to an mTOR (Mammalian target of rapamycin) gene having mutations in a nucleotide sequence or an mTOR protein having mutations in an amino acid sequence resulting from the mutations in the nucleotide sequence. Further, the present invention relates to a technique for diagnosing intractable epilepsy caused by malformations of cortical development using the gene or the protein.

Abstract: Disclosed herein are methods of treating a patient at risk of developing or having a neurofibromatosis or a sporadic schwannoma. In exemplary embodiments, the method involves administering to a subject in need an effective amount of a modulator of a target related to neurofibromatosis. Also disclosed are screening assays involving the implementation of Merlin-null Schwann cells, and to compounds identified using same.

Abstract: The present invention provides a pharmaceutical composition for treating infarction, such as myocardial infarction or cerebral infarction, with a method that is different from conventional therapeutic methods that use a thrombolytic agent or balloon therapy. An autophagy enhancer such as hsa-miR-145 is used as the active ingredient of the pharmaceutical composition for treating infarction. In particular, an autophagy enhancer such as hsa-miR-145 is used in a liposomal state as an active ingredient of the pharmaceutical composition for treating infarction.

Abstract: There is provided a composition for prophylaxis or treatment of vascular or valvular calcification including a dipeptidylpeptidase-4 (DPP-4) inhibitor. The DPP-4 according to one exemplary embodiment of the present disclosure is expressed at an increased level when blood vessels and valves are calcified, and the calcification decreases remarkably upon administration of the DPP-4 inhibitor. Therefore, the DPP-4 inhibitor can be useful in treatment or prophylaxis of blood vessels or valves.