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.

Abstract: Agents and methods for increasing dystrophin protein expression in muscle through blocking of specific microRNAs and microRNA binding sites on the dystrophin 3? untranslated region (miR-146a, miRNA-146b, miR-223, miR-320a, miR374a, and miR-382). Methods for increasing the amount of dystrophin useful for effective therapeutic intervention for Becker muscular dystrophy, Duchenne muscular dystrophy, and other disorders where loss of dystrophin from muscle causes pathology.

Abstract: The present invention relates to microRNAs (miRNAs) which are associated with cancer, particularly including hematologic malignancies, and particularly T-cell acute lymphoblastic leukemia (T-ALL), and to the assessment and modulation thereof in the treatment and management of cancer. The present invention is directed to methods and compositions for diagnosing and treating cancer, particularly T-ALL, by modulating miRNAs, and the use of miRNAs and antagonists thereof, particularly antagomirs, for predicting and assessing response to treatment, in assays for isolating and selecting antagonists, and as compositions for the treatment and management of cancer. Methods and compositions are provided for treatment or alleviation of cancer, particularly T-ALL, with antagonists/antagomirs of miRNAs, particularly one or more of miR-19b, miR-20a, miR26, miR92, miR148 and miR223.

Abstract: The present invention relates to siRNA hydrogel and a method for manufacturing the same, and more particularly, to siRNA hydrogel for targeted gene silencing, which is nano-structured for targeted gene silencing, and a method for manufacturing the same.

Abstract: ATM kinase is shown to regulate proteasome-mediated protein turnover through suppression of the expression of the ubiquitin-like protein ISG15 (Interferon Stimulated Gene 15). Silencing of the ISG15 pathway restored both the ubiquitin and autophagy pathways, and the UV-mediated degradation of their substrates in A-T cells. The ATM kinase negatively regulates the ISG15 pathway, and the constitutively elevated ISG15 pathway induces proteinopathy in A-T cells, and in A-T patients. These findings indicate that proteasome-mediated protein degradation is impaired in A-T cells due to elevated expression of the ISG15 conjugation pathway, which contributes to progressive neurodegeneration in A-T patients. The ISG15 pathway is a new target for both detection and treatment of A-T Inhibitors if ISG15 expression can be used to inhibit or attenuate neurodegeneration in A-T patients.

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: Provided are methods, compounds, and compositions for reducing expression of ApoCIII mRNA and protein for treating, preventing, delaying, or ameliorating Fredrickson Type I dyslipidemia/FCS/LPLD, in a patient. Such methods, compounds, and compositions increase HDL levels and/or improving the ratio of TG to HDL and reducing plasma lipids and plasma glucose in the patient, and are useful to treat, prevent, delay, or ameliorate any one or more of pancreatitis, cardiovascular disease, metabolic disorder, and associated symptoms.

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.

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: The present invention provides, among other things, oligonucleotide modulators of human 5?-HT2C receptor (HTR2C) and improved methods and composition for treating HTR2C-related diseases, disorders or conditions based on such modulators. In particular, oligonucleotides modulators according to the invention target specific regions in the Exon V/Intron V junction of the human HTR2C pre-mRNA and drive expression of HTR2C Vb splice isoform, leading to increased generation of non-edited strong HTR2C receptor and enhanced serotonin receptor activity.

Abstract: Provided are methods and compositions for modulating the differentiation of a myeloid derived suppressor cell (MDSC). In particular, described herein are miR-142 polynucleotides and miR-223 polynucleotides that can be used to modulate differentiation of MDSCs. Increased differentiation of a MDSC population, or cells within an MDSC population, can be achieved by increasing the miR-142 and/or miR-223 polynucleotides in a MDSC.

Abstract: The present invention provides methods for tumor treatment by administering an inhibitor of quiescin sulfhydryl oxidase 1 (QSOX1), compositions comprising such inhibitors, and methods for identifying such inhibitors.

Abstract: Methods and compositions are provided for treating HIV infection and for inhibiting HIV infection, and for identifying purinergic receptor antagonists or Panx 1 hemi-channel blockers useful therefor. The invention provides a method of treating a mammalian subject having an HIV infection, or suspected of having been exposed to HIV, comprising administering to the mammalian subject an amount of (i) an antagonist of a Panx 1 hemichannel, or (ii) of an inhibitor of a purinergic receptor, effective to inhibit (a) HIV fusion with a target cell, or (b) HIV replication, or (c) HIV entry into a target cell, or two or more of (a), (b) and (c).

Abstract: The invention provides a single-stranded nucleic acid molecule containing an expression inhibitory sequence that inhibits expression of a target gene, region (X), linker region (Lx), and region (Xc), wherein the linker region (Lx) is linked between the region (Xc) and the region (Xc), the region (Xc) is complementary to the region (X), at least one of the region (X) and the region (Xc) contains the expression inhibitory sequence, and the linker region (Lx) contains an atomic group derived from an amino acid. The single-stranded nucleic acid molecule can inhibit expression of the target gene.

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 present invention provides an alpha-methylacyl-CoA racemase binding aptamers. The present invention further provides a kit for detecting alpha-methylacyl-CoA racemase or cancer in a sample, which comprises the above-mentioned alpha-methylacyl-CoA racemase binding aptamers.

Abstract: Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.

Abstract: The present invention discloses multiple treatment regimens for vascular-related diseases and disorders. The present invention provides for methods of treating vascular-related disorders based on gene expression studies from samples collected from individuals having symptoms of vascular-related disorders. Additionally, methods are disclosed involving diagnostic techniques to focus treatment regimens. Finally, methods of treating vascular-related disorder involving targeting microRNAs are also disclosed.

Abstract: A method of ex-vivo increasing insulin content in beta cells or stem cells is disclosed. The method comprising contacting the beta cells or stem cells with an agent for downregulating an activity or expression of miR-7, thereby increasing the insulin content in the beta cells or stem cells.

Abstract: The present invention relates to novel compositions and therapeutic methods for the treatment of cancer, in particular malignant glioma. The compositions include antisense oligonucleotides or RNAs or vectors encoding them which reduce expression of downregulated in renal cell carcinoma (DRR) in tumor cells, and inhibit malignant glioma cell invasion.