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: This invention relates to long non-coding RNAs (lncRNAs), libraries of those ncRNAs that bind chromatin modifiers, such as Polycomb Repressive Complex 2, inhibitory nucleic acids and methods and compositions for targeting lncRNAs.

Abstract: This invention is about the selection and development of aptamers that specifically bound HSA and GHSA. HSA and GHSA are associated with diabetes mellitus. The length of selected aptamers are around 46-106 bases, in which aptamers against HSA are consisting of 46-106 bases and aptamers against GHSA are consisting of 49-71 bases. All selected aptamers against HSA and GHSA can be potentially applied for detection and monitoring of diabetes mellitus in combination with blood glucose and HbA1C level. They also can applied in the drug development and drug delivery system in the diabetes mellitus and Alzheimer disease. In addition, chemical or fluorescence labeled these aptamers can be used for study function and location of HSA and GHSA.

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 down-regulating the TLR2 gene or both the TLR2 gene and TLR4 gene.

Abstract: This invention relates to long non-coding RNAs (lncRNAs), libraries of those ncRNAs that bind chromatin modifiers, such as Polycomb Repressive Complex 2, inhibitory nucleic acids and methods and compositions for targeting lncRNAs.

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: Provided herein are compositions, method and uses for modulating IAP activity or for the treatment of cancer. The compositions comprise dual-targeting antisense oligonucleotides (dASO) for administration to a cancer cell, wherein the cancer cell may be characterized by elevated expression of one of more of BIRC6, cIAP1 or survivin. The cancer may be selected from one or more of: prostate cancer; childhood de novo acute myeloid leukemia; colorectal cancer; neuroblastoma; melanoma; and non-small cell lung cancer. The prostate cancer may be castration-resistant prostate cancer (CRPC).

Abstract: Molecular target for healing or treating wounds and, in particular chronic, human wounds, are described. The molecular target is PTPRK, or a protein 50% homologous therewith, and which retains the same activity as PTPRK protein. Further, methods and novel therapeutics are described for treating said wounds.

Abstract: A small interfering RNA is provided. The small interfering RNA consists of a passenger strand and a guide strand, wherein the sequence of the passenger strand comprises the sequence of SEQ ID NO. 7, and the sequence of the guide strand comprises the sequence of SEQ ID NO. 8, wherein the small interfering RNA is capable of inhibiting galectin-12 expression.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Sodium channel, voltage-gated, alpha subunit (SCNA), in particular, by targeting natural antisense polynucleotides of Sodium channel, voltage-gated, alpha subunit (SCNA). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of SCNA.

Abstract: This invention provides a novel delivery means that enables efficient delivery of an active ingredient to a target cell. Such novel delivery means is a liposome for topical administration that consists of dioleylphosphatidylethanolamine (DOPE), phosphatidylcholine, and cationic lipid, that is not modified with PEG, and that is free of cholesterol.

Abstract: This invention relates generally to segmented oligonucleotides capable of modulating gene expression. Specifically, the instant invention relates to segmented microRNA (miRNA) oligonucleotides, including segmented miRNA precursors and segmented pre-microRNAs. The invention also relates to compositions comprising such segmented oligonucleotides, as well as to methods of making and using such oligonucleotides for diagnosis and treatment of diseases associated or causally linked to aberrant levels or activities of gene expression, including aberrant levels of coding and/or non-coding RNA.

Abstract: Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

Abstract: Compositions comprising therapeutic oligonucleotide miR-3151 compounds that target the expression of genes associated with tumorigenesis or cell transformation are provided.

Abstract: The invention relates to modified siRNA compounds which down-regulate target gene expression, to pharmaceutical compositions comprising such compounds and to methods of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions.

Abstract: This invention provides compounds, compositions and methods for modulating the expression of human p21 using RNA interference. The RNA interference molecules can be used in methods for preventing or treating diseases such as malignant tumor. Provided are a range of siRNA structures, having one or more nucleotides being modified or chemically-modified. Advantageous structures include siRNAs with 2?-deoxy nucleotides located in the seed region, as well as other nucleotide modifications.

Abstract: Described herein are compositions and methods for the inhibition of miR-21 activity. The compositions have certain nucleoside modification patterns that yield potent inhibitors of miR-21 activity. The compositions may be used to inhibit miR-21, and also to treat diseases associated with abnormal expression of miR-21, such as fibrosis and cancer.

Abstract: Disclosed is providing a novel VEGF-binding aptamer whose affinity to VEGF is higher than those of known VEGF-binding aptamers. By an in silico maturation method starting from a known VEGF-binding aptamer, 4 kinds of aptamers whose affinities to VEGF are higher than that of the known VEGF-binding aptamer were prepared. By linking two molecules of an obtained aptamer to each other via a linker, an aptamer having an even higher affinity to VEGF was obtained. The polynucleotide of the present invention contains the base sequence of any one of SEQ ID NOs:1 to 4, and binds to vascular endothelial growth factor.

Abstract: An isolated nucleic acid agent is disclosed comprising a nucleic acid sequence which downregulates expression of a gene product of a Varroa destructor mite. Compositions comprising same and uses thereof are also disclosed.

Abstract: Disclosed herein are antisense compounds and methods for decreasing alpha-synuclein mRNA and protein expression. Also disclosed herein are methods for treating, preventing, and ameliorating neurodegenerative diseases in an individual in need thereof.