Abstract: The invention discloses a diagnosis of endometriosis, by determining a level of miR-199a-5p as a biomarker to diagnose endometriosis. The invention also discloses a treatment of endometriosis, by administering pre-miR-199a to a subject in need thereof in a dosage of 4 to 8 mg/per kilogram of body weight per 1 to 3 days for 4 weeks to inhibit the processes of endometriosis development.

Abstract: The invention relates to trans-acting ligand-responsive nucleic acids and uses thereof. In particular, a ligand responsive nucleic acid comprises an effector domain and an aptamer domain that is responsive to a ligand.

Abstract: Methods for malignant glioma diagnosis/prognosis using miR-138 as a prognostic biomarker and methods for treating malignant glioma and inhibiting glioma stem cell growth by suppressing miR-138. Also disclosed herein are pharmaceutical compositions for use in treating malignant glioma, prolonging survival of malignant glioma patients, or eliminating GSCs and in turn tumor growth, the pharmaceutical composition comprising an oligonucleotide that targets miR-138.

Abstract: The present invention provides compositions, pharmaceutical preparations, kits and methods for increasing expression of a gene product in a cell by contacting the cell with a small activating RNA (saRNA) molecule comprising a ribonucleic strand that is complementary to a non-coding nucleic acid sequence of the gene.

Abstract: The present disclosure provides a non-naturally occurring miRNA having a stem-loop structure comprising a scaffold derived from a first endogenous miRNA (e.g., miR-196a-2 or miR-204), a mature strand derived from a second endogenous miRNA, and a star strand sequence that is at least partially complementary to the mature strand sequence. The present disclosure also provides a non-naturally occurring miRNA having a stem-loop structure comprising a scaffold derived from an endogenous miRNA (e.g., miR-196a-2 or miR-204), a mature strand designed to be at least partially complementary to a target RNA, and a star strand sequence that is at least partially complementary to the mature strand sequence. The methods and compositions of the disclosure may be used to mediate gene silencing via the RNAi pathway.

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 invention relates to the therapeutic use of stabilized oligoribonucleotides as immune modulatory agents for immune therapy applications. Specifically, the invention provides RNA based oligoribonucleotides with improved nuclease and RNase stability and that have immune modulatory activity through TLR7 and/or TLR8.

Abstract: Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.

Abstract: The invention relates to oligonucleotides including at least one backbone modification and a pyrimidine-purine dinucleotide. The invention also relates to pharmaceutical compositions and methods of use thereof.

Abstract: The invention relates to methods and compositions for modulating viral replication through double-stranded RNA-mediated gene silencing (RNAi), wherein the antiviral methods and compositions preferentially target opposite strand replication intermediates of single-stranded RNA viruses.

Abstract: Provided is a higher quality aptamer having a binding activity to NGF. An aptamer binding to NGF, which satisfies the following (1) and (2): (1) containing the sequence represented by UGAAARAAACC (SEQ ID NO: 64) or CGAAMRAAACU (SEQ ID NO: 65), and (2) having a base length of not more than 73.

Abstract: The invention relates to oligonucleotides that bind to and induce skipping of exon 44 in the human dystrophin pre-mRNA, and methods of use.

Abstract: Compositions and methods for cancer treatment are discloses 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 augment tumor antigen expression, presentation, and processing through expression of the GM-CSF transgene.

Abstract: Amplification-based methods and kits for rapidly producing siRNA expression cassettes are provided. Also provided are methods for expressing amplified siRNA expression cassettes in cells.

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: The present invention concerns antagonists of the Golgi reassembly-stacking protein of 55 k Da (Grasp55), for use as a medicament, in particular for use in the treatment of cell adhesion molecules implicated diseases, notably, MCAM or JAMs implicated diseases. According to some embodiments, said antagonists of the Golgi reassembly-stacking protein of 55 k Da (Grasp55) may be used in the treatment of cancer, metastases, and inflammatory diseases. The present invention further provides methods for screening for compounds liable of treating or preventing such diseases.

Abstract: The present invention describes a flexible basestacking monomer that can be incorporated into an oligonucleotide or oligonucleotide analogue, as well as triplex forming oligonucleotides comprising the flexible basestacking monomer. Triplex forming oligonucleotides of the invention are capable of binding sequence specifically to doublestranded target nucleic acids and are therefore of interest for modulation of the activity of target nucleic acids and also detection of target nucleic acids.

Abstract: The present disclosure relates to the finding that microRNA-146 plays a role in modulating the development and function of the immune system. Immune cell development and function can be modulated by delivery of microRNA-146 (miR-146) or antisense miR-146 to target immune cells or precursor cells. For example, in some embodiments, activity and/or proliferation of certain immune cells is regulated by administering miR-146 oligonucleotides or anti-miR-146 oligonucleotides. In other embodiments, pro-inflammatory cytokine expression in immune cells is regulated by administering a miR-146 oligonucleotide or anti-miR-146. In further embodiments, methods of regulating macrophage activity using antisense miR-146 are provided. Additional methods and compositions for regulating immune system function and development using miR-146 are disclosed.

Abstract: The present disclosure relates generally to the field of nucleic acids and, more particularly, to aptamers capable of binding to toxins produced by Clostridium difficile; diagnostic kits and methods comprising such aptamers; and methods of making and using such aptamers.

Abstract: The invention provides an improved design for the construction of extensible nucleic acid-based, ligand-controlled regulatory systems, and the nucleic acid regulatory systems resulting therefrom. The invention contemplates improving the design of the switches (ligand-controlled regulatory systems) through the design of an information transmission domain (ITD). The improved ITD eliminates free-floating ends of the switching and the competing strands, and localizes competitive hybridization events to a contiguous strand of competing and switching strands in a strand-displacement mechanism-based switch, thereby improving the kinetics of strand-displacement. The improved regulatory systems have many uses in various biological systems, including gene expression control or ligand-concentration sensing.