Abstract: The invention relates to lipid formulated double-stranded ribonucleic acid (dsRNA) targeting a hepcidin antimicrobial peptide (HAMP) and/or HAMP-related gene, and methods of using the dsRNA to inhibit expression of HAMP and/or HAMP-related genes.

Abstract: The RNAi target sequences, which could be used for treating AIDS through targeting HIV. Based on the target sequences, recombinant expression vectors, packaging vectors and cells were constructed, which express siRNA and/or miRNA and/or ribozyme and/or antisense oligonucleotide for targeting HIV. And the applications of said recombinant expression vectors, packaging vectors and cells in preparing medicament for treating AIDS.

Abstract: Methods for heart regeneration are provided. The invention provided herein includes methods of modulating proliferation of cardiomyocytes using small molecules and micro RNAs. In embodiments, the methods provided may be used to increase proliferation or cardiomyocytes. Further provided are methods to be used for the treatment of myocardial infarction.

Abstract: The present invention is based, at least in part, upon discovery of a process for identifying phase changing peptides. Such phase changing peptides are capable of enhancing in vitro and in vivo delivery of oligonucleotides (e.g., dsRNAs) in lipidic, vesicular, micellar and/or naked oligonucleotide formulations.

Abstract: Disclosed herein are therapeutic methods for treating lung diseases, disorders and injury in a mammal, including treatment of acute respiratory distress syndrome (ARDS), acute lung injury, pulmonary fibrosis (idiopathic), bleomycin induced pulmonary fibrosis, mechanical ventilator induced lung injury, chronic obstructive pulmonary disease (COPD), chronic bronchitis, emphysema, bronchiolitis obliterans after lung transplantation and lung transplantation-induced acute graft dysfunction, including treatment, prevention or prevention of progression of primary graft failure, ischemia-reperfusion injury, reperfusion injury, reperfusion edema, allograft dysfunction, pulmonary reimplantation response, bronchiolitis obliterans after lung transplantation and/or primary graft dysfunction (PGD) after organ transplantation, in particular in lung transplantation, comprising downregulating the TLR2 gene or both the TLR2 gene and TLR4 gene.

Abstract: The present invention relates to a method for treating a cancer including a combination of a treatment by a nucleic acid molecule mimicking double strand breaks with hyperthermia.

Abstract: The present disclosure is directed to compositions and methods for detecting elevated microRNA U6 concentrations in serum relative to a standard microRNA (e.g. SNORD44) as a diagnostic indicator of metastatic disease. In one embodiment the methods of the present disclosure are used to diagnose the existence of, or assess the risk of, breast cancer in an individual.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Mylip/Idol gene, and methods of using such dsRNA compositions to inhibit expression of Mylip/Idol.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting one or more EGLN genes, EGLN1, EGLN2 and/or EGLN3 and methods of using such dsRNA compositions to inhibit expression of these genes.

Abstract: The invention relates to methods and biomarker for evaluating cancer metastasis, pharmaceutical composition for inhibiting cancer metastasis, and method for analyzing secretome. By combining a hollow fiber cartridge (HFC) culture system with quantitative proteomics technology, cancer metastasis-related secrectomes can be found. Furthermore, this is the first time to use PARK7 as a biomarker for judging the process of non-small cell lung cancer.

Abstract: The present invention relates to anti-sense oligonucleotides (AONs) used to induce exon 9 skipping in IL-23R? gene. Exon 9 skipping of the IL23R? gene ultimately causes specific induction of a novel soluble truncated IL-23R? (?9) protein, characterized by a lack in a transmembrane domain and has a unique eight (8) amino acids (GLKEGSYC) at its C-terminus end as a result of frame-shift. The present invention provides a utility application of the use of AONs to induce production of a ?9 protein which inhibits IL-23R-mediated cell signaling. More particularly, ?9 protein blocks STAT3 formation as well as Th17 maturation. There is provided a therapeutic application of AONs in treating a mammal such as a human patient inflicted with Crohn's disease.

Abstract: The invention relates to a method for the immune activation of NK cells by the reduction or inhibition of the Cbl-b function in said cells. This stimulates the congenital immune system and thus permits the therapy of appropriate diseases.

Abstract: A method of diagnosing and treating a glioma or a breast cancer is disclosed. The method of diagnosing comprises analyzing an amount or activity of heterogeneous nuclear ribonucleoprotein A2/B1 (hnRNP A2/B1) in a brain or breast cell sample of the subject, wherein an up-regulation in an amount or activity of hnRNP A2/B1 beyond a predetermined threshold with respect to a control cell sample is indicative of the breast cancer or glioma. The method may also be used for staging the cancer and for predicting patient's prognosis of survival. The method of treating glioblastoma and metastatic breast cancer includes inhibiting the expression and/or activity of hnRNP A2/B1.

Abstract: The present invention is directed to RNA interference (RNAi) molecules targeted against a Huntington's disease nucleic acid sequence, and methods of using these RNAi molecules to treat Huntington's disease.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the TMPRSS6 gene, and methods of using such dsRNA compositions to inhibit expression of TMPRSS6.

Abstract: Certain embodiments are directed to compounds and compositions targeted to human androgen receptor (AR) for inhibiting androgen receptor levels in a cell, which can be useful for methods of treating cancer and inhibiting cancer cell growth or proliferation.

Abstract: Disclosed herein are methods relating to inhibiting or reducing proliferation of a cancer cell, for treating cancer in a subject in need of treatment, predicting the risk of progression of cancer to a more aggressive cancer, and screening for cancer in a subject, that comprise detecting and/or decreasing the levels of SUMO conjugated proteins and detecting and/or interfering with SUMO conjugation.

Abstract: The invention relates to the diagnostic and therapeutic uses of a miRNA molecule or an equivalent thereof wherein a source of said miRNA molecule or equivalent thereof comprises at least 80 nucleotides and comprises a motif having at least 98% identity with the motif represented by SEQ ID NO:1 or a source thereof in a disease and condition associated with EMT (Epithelial to Mesenchymal Transition).

Abstract: The present invention relates to methods of identifying oligonucleotides capable of modulating the immune system in a mammalian subject, comprising analysis of which tertiary structural type said oligonucleotide adopts, in phosphate-buffered saline solution. Further, the invention provides oligonucleotides identifiable by the methods of the invention and to their use in methods of treating diseases, such as inflammatory diseases, autoimmune diseases, infectious diseases, neurodegenerative diseases and cancer.

Abstract: Described herein are compositions and methods for the inhibition of miR-122 activity. The compositions have certain nucleoside modifications that yield potent inhibitors of miR-122 activity. The compounds may comprise conjugates to facilitate delivery to the liver. The compositions may be administered to subjects infected with hepatitis C virus, as a treatment for hepatitis C virus and related conditions.