Abstract: The invention relates to a method for influencing the miR-92 expression in a cell, comprising the following steps: (a) providing a cell; and (b1) reducing the miR-92 expression in the cell in order to promote the vascularization or vessel repair by introducing an antisense molecule against miR-92 into the cell, or (b2) increasing the miR-92 expression in the cell for an inhibition of the tumor angiogenesis by introducing a construct into the cell, wherein said construct includes an expressible miR-92 sequence. Furthermore, the invention relates to a pharmaceutical composition, comprising an agent for reducing the miR-92 activity or expression in a cell in the form of an antisense molecule against miR-92, or an agent for increasing the miR-92 expression in a cell in the form of a construct for expressing miR-92.

Abstract: The present disclosure provides compositions and methods for treating or reducing the risk of pancreatic cancer by administering compounds capable of inhibiting the expression or activity of RUNX3.

Abstract: A unit structure-type pharmaceutical composition includes at least one nucleic acid, such as siRNA, electrostatically bound to at least one block copolymer having a cationic polyamino acid segment and a hydrophilic polymer chain segment. The negative charge(s) of the nucleic acid are counterbalanced, at least substantially, by the positive charge(s) of the cationic polyamino acid segment such that the pharmaceutical composition is electrically neutral or nearly electrically neutral. Further, the nucleic acid is covered with the hydrophilic polymer chain segment(s). The at least one block copolymer thereby improves the blood retention capability of the nucleic acid(s).

Abstract: A method for preventing or treating epilepsy or status epilepticus, or a brain-related disorder which is characterized by precipitation of seizures, in an individual. The method comprises the step of treating the individual with an agent that inhibits the activity of miR-134 in the individual. The agent is delivered to the brain of the individual.

Abstract: Compositions and methods for cancer treatment are disclosed herein. More specifically, the present invention describes an autologous cancer vaccine genetically modified for Furin knockdown and GM-CSF expression. The vaccine described herein attenuates the immunosuppressive activity of TGF-? through the use of bi-functional shRNAs to knock down the expression of furin in cancer cells, and to segment tumor antigen expression, presentation, and processing through expression of the GM-CSF transgene.

Abstract: The present invention concerns methods and compositions regarding one or more microRNAs or variants thereof that are provided to an individual for a variety of medical treatments, including sensitization to cancer therapy or prevention of a cancer to become sensitized to a cancer therapy. In specific embodiments, the microRNAs include miR-520a (including at least miR-520a-3p and miR-520-5p), miR-520g, miR-520h, and functional variants thereof. In some embodiments, the cancer is ovarian cancer, and in particular embodiments, the cancer therapy is platinum-based chemotherapy.

Abstract: The present invention provides gapped oligomeric compounds comprising at least one 5?-substituted P-D-2?-deoxyribonucleoside in the gap region. Certain such gapped oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited to, nucleic acids in a cell. The oligomeric compounds provided herein have improved properties such as selectivity, potency and improved proinflammatory profile. In certain embodiments, hybridization results in modulation of the amount of activity or expression of the target nucleic acid in a cell.

Abstract: The present invention relates to methods and compositions for enhancing neuronal connectivity. It is based, at least in part, on the discovery of a protein, termed “Mirta22,” that inhibits the formation of structures which create connections between neurons. It is further based, in part, on the discovery that inhibiting Mirta22 activity by short hairpin RNA was able to restore these structures. Mirta22 was discovered in experiments relating to 22q11 microdeletions, which have been linked to schizophrenia. Accordingly, the present invention provides for methods of treating schizophrenia comprising administering an agent that inhibits Mirta22 activity. It may also be used in the treatment of other disorders that would benefit from enhanced neural connectivity, including learning and memory disorders.

Abstract: The invention relates to methods of inhibiting the growth or proliferation of a cell, the method comprising reducing the expression or activity of at least one gene in the cell selected from the group consisting of BLOC1S1, CDC2L1, CNOT1, DDX54, EIF3I, FANCG, FBP1, IER2, KIF1A, LCK, NR2F1, PNRC1, POLR2A, POLR2B, POLR2C, PRPF6, PSMB4, PSMC5, PSMD1, RPS2, SCNN1A, SF3A3, TAF2, TOB1 and TSC22D4.

Abstract: Described herein are polynucleotides associated with prostate and lung cancer. The polynucleotides are miRNAs and miRNA precursors. Related methods and compositions that can be used for diagnosis, prognosis, and treatment of those medical conditions are disclosed. Also described herein are methods that can be used to identify modulators of prostate and lung cancer.

Abstract: Described herein are novel polynucleotides associated with prostate and lung cancer. The polynucleotides are miRNAs and miRNA precursors. Related methods and compositions that can be used for diagnosis, prognosis, and treatment of those medical conditions are disclosed. Also described herein are methods that can be used to identify modulators of prostate and lung cancer.

Abstract: The present invention relates to methods and pharmaceutical compositions for the treatment of Erythropoietic Protoporphyria. In particular, the present invention relates to a method for increasing the amount of functional FECH in a erythroid cell carrying the hypomorphic allele IVS3 48C/T (rs2272783) in trans to a deleterious mutation in the FECH gene comprising the step of consisting of bringing the erythroid cell into contact with at least one antisense oligonucleotide (ASO) comprising the sequence as set forth by SEQ ID NO: 2 (5? gcagcctgagaaatgtttt 3?) to prevent splicing of the cryptic exon inserted into the mutant IVS3 48C/T (rs2272783) FECH mRNA.

Abstract: Certain embodiments are directed to methods and compounds for inhibiting UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A). Such methods and compounds are useful for inducing expression of paternal UBE3A in cells and animals.

Abstract: The invention provides multifunctional supramolecular self-assembled nanoparticles (SSNPs) comprising a set of rationally designed components that collectively facilitate efficient intestinal absorption of siRNA. The nanoparticles can induce potent TNF-? silencing in macrophages. Single gavage of SSNPs in mice depleted systemic TNF-? production at an siRNA dose as low as 50 ?g/kg, and protected the mice from lipopolysaccharide-induced hepatic injury.

Abstract: The present invention provides compositions for RNA interference and methods of use thereof. In particular, the invention provides single-stranded small interfering RNAs. Functional and genomic and proteomic methods are featured. Therapeutic methods are also featured.

Abstract: Provided herein are fluoropyrimidine-modified RNA aptamers capable of binding CCR5. The compositions and methods provided herein are, inter alia, useful for the delivery of anti-viral drugs (e.g., siRNAs) and preventing HIV entry into a target cell.

Abstract: Aspects of the invention relate to methods and compositions for characterizing or modulating the expression of metabolic mesenchymal genes. In some embodiments, methods for assessing the expression of metabolic mesenchymal genes and related gene signatures are provided that are useful for cancer classification, prognosis, diagnosis, or treatment selection.

Abstract: Provided is a composition for inducing tumor cell senescence and a method for inducing tumor cell senescence using the same. The composition can inhibit a PAPSS2 gene in a tumor cell to thereby induce senescence of the tumor cell, and if the composition is used concurrently with irradiation of the tumor cell, the composition can improve the sensitivity of the tumor cell to radiation.

Abstract: The present invention is directed to a method and use of RNA interference (RNAi) for the transient, reversible and controlled opening of the tight junctions of the blood brain barrier and/or the blood retinal barrier. This method may be used in the treatment of many diseases and disorders which require the opening of the blood brain barrier and/or blood retinal barrier. Such methods generally involve the use of an RNAi-inducing agent, such as siRNA, miRNA, shRNA or an RNAi-inducing vector whose presence within a cell results in production of an siRNA or shRNA, targeting tight junction proteins to open the blood brain barrier and/or blood retinal barrier.

Abstract: The present invention provides methods of preparing an oligonucleotide, nucleoside or nucleoside analog for selective introduction into a subject's cells, the method comprising (1) selecting a targeted aptamer, internalizing nucleic acid or tumor-homing nucleic acid via iterative rounds of selection, and (i) hybridizing it to an oligonucleotide, (ii) replacing one or more nucleotide with a nucleoside or nucleoside analog, or (iii) synthesizing the it with one or more nucleoside or nucleoside analogs; or (2) preparing a naive combinatorial aptamer, internalizing nucleic acid or tumor-homing nucleic acid prodrug library, and running iterative rounds of selection for the cells. The present invention also provides the agent, the pharmaceutical composition, and methods of treating or preventing cancer and/or viral infection, the method comprising administration of the oligonucleotide, nucleoside or nucleoside analog for selective introduction into a subject's cells.