Abstract: The present invention provides a method of inducing insulin production in a cell by up-regulating a target gene involved in insulin production in said cell using an saRNA (short activating ribonucleic acid) which specifically targets a target antisense RNA transcript present in said cell.

Abstract: A treatment of cancer, particularly the treatment of neuroblastoma. Indeed, the discloses that a particular long non-coding RNA (lncRNA) is specifically up-regulated in neuroblastoma cells as compared to other cancer cells and that high expression of the lncRNA correlates with a lower survival probability. Inhibition of this lncRNA in neuroblastoma cells leads to a reduction in cell growth and induction of apoptosis and is a novel therapeutic strategy in the treatment of neuroblastoma.

Abstract: This invention relates to an agent that modulates the activity of a micro ribonucleic acid (miRNA), said miRNA being selected from the group consisting of miRNA 429-3p, miRNA 29c-3p, miRNA 29b-3p, miRNA 200a-3p, miRNA 200b-3p, miRNA 200c-3p, miRNA 141-3p, miRNA 15a-5p, miRNA 15b-5p, miRNA 16-5p, miRNA 424-5p, miRNA 497-5p, miRNA 615-3p, miRNA 451a-5p and miRNA 542-5p, for use in the treatment of HER2-dependent cancer.

Abstract: The present disclosure relates to compositions and methods for inhibiting nonsense-mediated mRNA decay in a gene-specific manner, for example in the treatment of diseases or disorders caused by nonsense mutations.

Abstract: Compositions, recombinant viruses, and recombinant viral vectors for inhibiting sarcolipin (SLN) expression or activity in a cell and for preventing or treating Duchenne Muscular Dystrophy (DMD) in a subject (e.g., a human patient having or predisposed to having DMD) and in some embodiments additionally cardiomyopathy, include a therapeutically effective amount of an inhibitor of SLN. Methods of using these compositions, recombinant viruses, and recombinant viral vectors are also described herein. These compositions, recombinant viruses, and recombinant viral vectors and methods of use provide novel therapies for DMD and associated cardiomyopathy based on the reduction of SLN expression and/or activity.

Abstract: Methods of inhibiting influenza A virus in a sample are provided. Aspects of the methods include contacting a sample comprising viral RNA (vRNA) having a PSL2 motif with an effective amount of an agent that specifically binds the PSL2 motif to inhibit the influenza A virus. Also provided are methods of treating or preventing influenza A virus infection in a subject. Also provided are methods for screening a candidate agent for the ability to inhibit influenza A virus in a cell, the method comprising: contacting a sample with a candidate agent; and determining whether the candidate agent specifically binds to the PSL2 motif of vRNA. Also provided are compounds and pharmaceutical compositions comprising an oligonucleotide sequence complementary to a PB2 vRNA region that find use in the subject methods.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.

Abstract: A method of producing an aptamer selectively binding a non-canonical structure of a target nucleic acid molecule includes the steps of: incubating a plurality of nucleic acid sequences with an enantiomer of the non-canonical structure under suitable conditions to obtain one or more candidate nucleic acid sequences binding to the enantiomer of the non-canonical structure, purifying and amplifying the one or more candidate nucleic acid sequences; repeating said incubating, purifying and amplifying steps for a predetermined number of cycles under different conditions; and producing an enantiomer for selected amplified candidate nucleic acid sequence to obtain the aptamer capable of selectively binding the non-canonical structure of the target nucleic acid molecule. An aptamer selectively binding to a non-canonical structure of a nucleic acid molecule or its enantiomer, the aptamer comprising a sequence of SEQ ID NO: 11; as well as uses of the aptamer or its enantiomer.

Abstract: Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a). In certain embodiments, the apo(a) targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting apo(a) for use in decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) and/or Lp(a). Certain diseases, disorders or conditions related to apo(a) and/or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.

Abstract: The present invention relates to a compound comprising a modified saccharide moiety conjugated to a nucleic acid. The compound is useful in medicine for RNA interference therapy or for research and diagnostic purposes. In particular, the compound is useful in treating liver disease.

Abstract: An oligonucleotide that inhibits expression of an OLMALINC nucleic acid molecule, such as a small inhibitory RNA (siRNA) molecule, can be used for inhibiting the expression of OLMALINC in a subject. Methods of assaying for OLMALINC in a tissue sample can be used for detecting a disorder associated with obesity and/or type 2 diabetes in a tissue sample obtained from a subject. A method of ameliorating symptoms associated with obesity and/or type 2 diabetes comprises administering to a subject in need thereof an effective amount of an oligonucleotide of the invention or an antibody or equivalent thereof that specifically binds to and inactivates an OLMALINC nucleic acid molecule. Representative examples of a disorder associated with obesity and/or type 2 diabetes include, but are not limited to, a disorder of appetite, glycemia, body weight, liver steatosis, NASH, NAFLD, or a lipid disorder.

Abstract: The present invention provides nucleic acid aptamers binding to the Intercellular Adhesion Molecule-1 (ICAM-1), derivatives and conjugates thereof and their use as diagnostic tools, particularly for the imaging of organs and tissues expressing ICAM-1, or as therapeutic agents for prevention or treatment of ICAM-1-related diseases.

Abstract: The presently disclosed subject matter relates to the administration of a KMT2D inhibitor for the treatment of a cancer. The present invention is based on the discovery that upon PI3K inhibition, KMT2D activity is upregulated, resulting in an increase in the expression of genes involved in breast cancer cell proliferation and tumor growth. Accordingly, the present invention provides methods for treating a subject that has cancer by administering a therapeutically effective amount of an KMT2D inhibitor.

Abstract: A composition for treating cancer is disclosed. The composition includes a lentiviral particle and an aminobisphosphonate drug. The lentiviral particle is capable of infecting a target cell, such as a cancer cell, and includes an envelope protein optimized for targeting such target cell and a viral vector. The viral vector includes a small RNA optimized to target an FDPS mRNA sequence. The aminobisphosphonate drug includes zoledronic acid.

Abstract: The present invention provides a novel molecule that can be used for detection of sIgA. The sIgA-binding nucleic acid molecule of the present invention is characterized in that it binds to secretory immunoglobulin A (sIgA) with a dissociation constant of 37.7 nM or less, and preferably includes a polynucleotide consisting of any of base sequences of SEQ ID NOs: 1 to 12 or a partial sequence thereof, for example. According to the sIgA-binding nucleic acid molecule of the present invention, it is possible to detect sIgA in saliva.

Abstract: The present invention, at least in part, relates to the discovery of efficacious delivery of an RNAi agent (in preferred aspects of the invention, an siRNA) to a transplantable tissue. Organ rejection, transplantation-mediated transmission of viral infection, and triggering of apoptosis in transplanted tissues can each be minimized by the methods and compositions of the instant invention. The RNAi agent(s) of the instant invention can be delivered as “naked” molecules, or using liposomal and other modes of delivery, to transplantable tissues. Such delivery can occur via perfusion of the RNAi agent in solution through the vasculature of a whole or partial organ; or tissues including transplantable cells and cell lines may be bathed, injected or otherwise treated with RNAi agents. Preferred transplantable tissues include, for example, pancreas, liver, kidney, heart, lung, and all cells and cell lines derived from such tissues (e.g., pancreatic islet cells that may, e.g.

Abstract: The present invention relates to a compound inhibiting the expression and/or the activity of a long non-coding RNA (lncRNA) selected from GADLOR 1 and GADLOR 2 for use in treating or preventing cardiac remodelling, wherein GADLOR 1 comprises or consists of a nucleic acid sequence selected from the group consisting of SEQ ID NOs 1 to 3 and sequences being at least 75% identical thereto, and GADLOR 2 comprises or consists of a nucleic acid sequence selected from the group consisting of SEQ ID NOs 4 to 6 and sequences being at least 75% identical thereto.