Abstract: The invention relates to a RNA interference triggers for inhibiting the expression of an AAT gene through the mechanism of RNA interference. The invention also relates to a pharmaceutical composition comprising the AAT RNAi trigger together with an excipient capable of improving delivery of the RNAi trigger to a liver cell in vivo. Delivery of the AAT RNAi trigger to liver cells in vivo provides for inhibition of AAT gene expression and treatment of alpha 1-antitrypsin deficiency and associated diseases.

Abstract: The present invention relates to a therapeutic agent for immune cell migration-caused disease and a method for screening the same and, more particularly, to a pharmaceutical composition comprising a KRS inhibitor (or expression or activity inhibitor) as an effective ingredient for preventing or treating an immune cell migration-related disease, a method for controlling the migration of immune cells by regulating a level of KRS in immune cells, a cell membrane site-specific moiety level of KRS or the migration of KRS to the cell membrane, and a method for screening a therapeutic agent for immune cell migration-caused disease, using KRS. According to the present invention, the migration of immune cells can be controlled by means of KRS, which can find very useful applications in the prevention, alleviation, and treatment of immune cell migration-related disease.

Abstract: Methods and compositions involving exosomes or lipid nanovesicles are provided. For example, certain aspects relate to compositions comprising exosomes obtained from cells that have been induced to undergo oxidative stress or stimulated. Furthermore, some aspects of the invention provide methods of treating a subject at risk or having a demyelinating disorder using the compositions.

Abstract: The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model.

Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting EZH2 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with EZH2.

Abstract: This invention relates to compounds, compositions, and methods useful for reducing lactate dehydrogenase target RNA and protein levels via use of ds RNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Abstract: The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of combinations of immunomodulatory polynucleotides (e.g., mRNAs) encoding an immune response primer polypeptide (e.g., an interleukin 23 (IL-23) polypeptide or an interleukin 36? (IL-36-gamma) polypeptide), and an immune response co-stimulatory signal polypeptide (e.g., an OX40L polypeptide).

Abstract: The invention provides a single-stranded oligonucleotide (ssON) for use in preventing or treating an influenza virus infection in a subject, wherein the single-stranded oligonucleotide is one that inhibits endocytosis. The invention also provides a single-stranded oligonucleotide (ssON) that comprises or consists of a polynucleotide having a sequence sharing at least 60% sequence identity with a sequence of any one of SEQ ID NOs: 13-21 listed in Table 1, or a complementary sequence thereof.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Collagen gene, in particular, by targeting natural antisense polynucleotides of a Collagen gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Collagen genes.

Abstract: The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting a xanthine dehydrogenase (XDH) gene, and methods of using such double stranded RNAi agents to inhibit expression of an XDH gene and methods of treating subjects having an XDH-associated disease.

Abstract: The present disclosure provides compositions with a modulating gene expression and methods for modulating transcription.

Abstract: The present disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human interleukin-12 (IL12), functional fragments thereof, and fusion proteins comprising IL12. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the disclosure provides sequence-optimized polynucleotides comprising nucleotides encoding the polypeptide sequence of human IL12, or sequences having high sequence identity with those sequence optimized polynucleotides.

Abstract: There are provided polynucleotides that is able to bind to and inhibit the long non coding RNA transcript SCAT7. These polynucleotides can be used for the treatment of cancer. Expression analysis of SCAT7 can be used for diagnosis of cancer.

Abstract: Provided are an EGFR gene expression-suppressing siRNA, a precursor sequence of same, and uses thereof.

Abstract: Provided are a therapeutic and/or prophylactic agent for a lung disease and a method for screening for the therapeutic and/or prophylactic agent. Provided are a therapeutic and/or prophylactic agent for a lung disease comprising an Arid5A inhibitor as an active ingredient and a method for screening for the therapeutic and/or prophylactic agent.

Abstract: The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to modified oligonucleotides which can be applied in method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.

Abstract: The present invention provides a novel pharmaceutical composition for treating and/or preventing cancer comprising, as an active ingredient, a polynucleotide derived from various miRNAs associated with cancer, a combination drug of the pharmaceutical composition and another antitumor agent, and a method for treating or preventing cancer in a subject having the cancer using the pharmaceutical composition or the combination drug. The present invention relates to a pharmaceutical composition for treating and/or preventing cancer comprising, as an active ingredient, a polynucleotide comprising the nucleotide sequence as set forth in SEQ ID NO: 1, or a nucleotide sequence comprising a substitution of one nucleotide in the nucleotide sequence as set forth in SEQ ID NO: 1 (wherein when at least a part of the polynucleotide is DNA, uracil in a region corresponding to the DNA in the nucleotide sequence is replaced with thymine).

Abstract: The present invention aims to provide an antisense oligonucleic acid with reduced hepatotoxicity. The antisense oligonucleic acid according to the present invention is characterized in that it has a base length of not less than 7 nt and not more than 30 nt, wherein nucleic acid residues of not less than 1 nt and not more than 5 nt respectively from the both terminals are 2?,4?-bridged nucleic acids, 2?,4?-non-bridged nucleic acid residue(s) is(are) present between the above-mentioned both terminals, and one or more bases in the nucleic acid residue(s) of the above-mentioned 2?,4?-non-bridged nucleic acid residue(s) is/are modified.

Abstract: This invention provides a method for the in vivo delivery of oligonucleotides. The invention utilizes the presence of one or plurality of HES linked to an oligonucleotide to deliver a nucleic acid sequence of interest into the cytoplasm of cells and tissues of live organisms. The delivery vehicle is nontoxic to cells and organisms. Since delivery is sequence-independent and crosses membranes in a receptor-independent manner, the delivered oligonucleotide can target complementary sequences in the cytoplasm as well as in the nucleus of live cells. Sequences of bacterial or viral origin can also be targeted. The method can be used for delivery of genes coding for expression of specific proteins, antisense oligonucleotides, siRNAs, shRNAs, Dicer substrates, miRNAs, anti-miRNAs or any nucleic acid sequence in a living organism. The latter include mammals, plants, and microorganisms such as bacteria, protozoa, and viruses.

Abstract: RNAi constructs directed to MAP4K4 that demonstrate unexpectedly high gene silencing activities, and uses thereof are disclosed. The blunt-ended constructs have a double-stranded region of 19-49 nucleotides. The constructs have selective minimal modifications to confer an optimal balance of biological activity, toxicity, stability, and target gene specificity. For example, the strands may be modified (e.g., one or both ends of the sense strand is modified by 2?-O-methyl groups), such that the construct is not cleaved by Dicer or other RNAse III, the antisense strand may also be modified by a 2?-O-methyl group at the penultimate 5?-end nucleotide to greatly reduce off-target silencing.