Abstract: Described herein are compounds useful for the treatment and investigation of diseases, methods for the prediction of in vivo toxicity of compounds useful for the treatment and investigation of diseases, and methods of discovering and identifying compounds useful for the treatment and investigation of diseases that have reduced in vivo toxicity.

Abstract: The present invention relates to RNAi molecules, and compositions thereof, comprising a 2? internucleoside linkage connecting the nucleotide at position 1 and the nucleotide at position 2 at the 5? end of the antisense strand. Specifically, the invention relates to single- and double-stranded short interfering nucleic acid (siNA) molecules that are capable of mediating RNA interference comprising 5? modified nucleotides that comprise, among other potential modifications, a 2? internucleoside linkage. The invention further relates to 5? modified nucleotides used as reagents to generate the RNAi molecules of the invention and methods of using the disclosed RNAi molecules.

Abstract: The invention relates to antisense oligonucleotidic sequences (ODN) against Smad7 suitably modified, and their uses in medical field as therapeutic biological agents, in particular in the treatment of chronic inflammatory bowel disease, such as Crohn's disease and ulcerative colitis.

Abstract: The present invention is directed to a storage-stable formulation of long-chain RNA. In particular, the invention concerns a dry powder composition comprising a long-chain RNA molecule. The present invention is furthermore directed to methods for preparing a dry powder composition comprising a long-chain RNA molecule by spray-freeze drying. The invention further concerns the use of such a dry powder composition comprising a long-chain RNA molecule in the preparation of pharmaceutical compositions and vaccines, to a method of treating or preventing a disorder or a disease, to first and second medical uses of such a dry powder composition comprising a long-chain RNA molecule and to kits, particularly to kits of parts, comprising such a dry powder composition comprising a long-chain RNA molecule.

Abstract: A single stranded oligonucleotide containing two or more acyl-amino-LNA or hydrocarbyl-amino-LNA nucleotide monomers, in which other nucleotide monomers can be DNA, RNA or chemically modified nucleotide monomers; in which the monomers of the oligonucleotide are linked by phosphodiester linkages and/or phosophorothioate linkages and/or phosphotriester linkages, in which the acyl and hydrocarbyl groups of the acyl-amino-LNA and hydrocarbyl-amino-LNA monomers are optionally substituted and thus optionally contain one or more hydroxyl group(s), amino group(s), thio group(s), oxo group(s), alkylthio group(s), ether group(s), and/or thiol (mercapto) group(s), and in which the acyl and hydrocarbyl groups of the acyl-amino-LNA and hydrocarbyl-amino-LNA monomers are linear or branched chains, cyclic or a combination of both, provided that the total number of carbon atoms in each acyl and hydrocarbyl group is less than 30.

Abstract: The present invention includes compositions and methods for treating arthritic joints found in patients with autoinflammation, e.g., systemic onset juvenile idiopathic arthritis, by administering at the site of inflammation a therapeutically effective amount of at least one agent that reduces or blocks the bioavailability of interleukin-1?.

Abstract: Provided herein are, inter alia, nucleic acid compounds useful for targeting CTLA-4-expressing cells and modulating cell activity of the CTLA-4-expressing cells. The compositions provided herein may be part of pharmaceutical compositions and may be used for treatment of cancer, inflammatory diseases, infectious diseases or metabolic diseases.

Abstract: Disclosed is a gene/carrier complex for preventing or treating inflammatory diseases, including tumor necrosis factor-? converting enzyme (TNF-? converting enzyme, TACE) shRNA and a nonviral gene carrier, wherein the nonviral gene carrier includes an acetate of disulfide-linked poly(oligo-arginine) or a TFA salt of poly(oligo-aspartic acid)poly(oligo-arginine).

Abstract: The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.

Abstract: Described herein are cell-selective mRNA constructs that can contain a RNA of interest and one or more miRNA targets. The cell-selective mRNA constructs described herein can be used to express an RNA of interest to a cell in a cell-selective manner.

Abstract: The invention relates to polynucleotide agents, e.g., antisense polynucleotide agents, targeting an angiotensinogen (AGT) gene, and methods of using such polynucleotide agents to inhibit expression of AGT and to treat subjects having an AGT-associated disease, e.g., hypertension.

Abstract: Provided herein are methods of treating and/or preventing endometriosis or symptom thereof, assays for diagnosing/prognosing endometriosis, compositions and formulations for treating and/or preventing endometriosis or symptom thereof, and populations of endometiotic cells, including life-extended populations of cells.

Abstract: Methods for introducing nucleic acids to cells via exosomes for use in gene modulation and therapy, such as for gene silencing and to introduce genetic material into cells to compensate for abnormal genes or to induce or repress a process in the recipient cell.

Abstract: The present invention provides methods for retaining and expressing physiologically active substances in a target tissue-specific-manner, by administering the physiologically active substances to target submucous tissue. Specifically, the present inventors demonstrated that, when physiologically active substances were directly administered into submucous tissues without using a carrier, the physiologically active substances were effectively and safely retained at the administration sites over long periods without loss and diffusion, and produced the effect acting in a reservoir-like fashion. The physiologically active substances administered as described above were demonstrated to produce the therapeutic effect without having an influence on organs other than the administered organ.

Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.

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

Abstract: The invention relates to color-changing eye drops for early screening Alzheimer's disease and an application thereof. The color-changing eye drop for early screening Alzheimer's disease comprises 1 pg/ml to 10 ng/ml of A?42 aptamer-gold nanoparticles (AuNPs) and a pharmaceutically acceptable carrier or vehicle thereof, which can be used to mix with a tear sample of a test subject to analyze a color change of the color-changing eye drop for determining the concentration proportion of A?40:A?42 contained in the tear sample of the test subject.

Abstract: Disclosed herein are antisense compounds and methods for decreasing SOD-1 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate SOD-1 associated diseases, disorders, and conditions. Such SOD-1 associated diseases include amyotrophic sclerosis (ALS).

Abstract: The present invention provides means for evaluating harmfulness of a chemical substance before occurrence of cell death, that is, more quickly and sensitively compared to conventional methods wherein the remaining viable cell count is determined using a reductive coloring reagent after a period of time required for occurrence of cell death due to the chemical substance. A double-stranded RNA probe comprising an RNA strand labeled with a fluorescent dye A that emits fluorescence, and an RNA strand labeled with a fluorescent dye B that quenches emission from a fluorescent dye in the vicinity thereof, wherein the fluorescence is quenched in a double-stranded state due to occurrence of fluorescence resonance energy transfer (FRET) between the two kinds of fluorescent dyes.

Abstract: Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels.