Abstract: The present disclosure relates to branched and linear chimeric compounds containing both nucleic acid and non-nucleic acid moieties, as well as to polynucleotides. The present disclosure also relates to uses thereof for stimulating an immune response, and to methods for preparation of the branched chimeric compounds.

Abstract: The present invention is directed to methods and compositions for identifying targets for induction of self-tolerance and treatment of autoimmune disease.

Abstract: Provided is a novel double-stranded ribonucleic acid for suppressing expression of complement C5. A double-stranded ribonucleic acid comprising a combination of sense strand and antisense strand, wherein the combination of the sense strand and antisense strand are selected from the group consisting of combinations: SEQ ID NO: 13/14, SEQ ID NO: 159/160, SEQ ID NO: 115/116, SEQ ID NO: 117/118, SEQ ID NO: 119/120, SEQ ID NO: 121/122, SEQ ID NO: 123/124, SEQ ID NO: 125/126, SEQ ID NO: 127/128, SEQ ID NO: 129/130, SEQ ID NO: 131/132, SEQ ID NO: 133/134, SEQ ID NO: 137/138, SEQ ID NO: 139/140, SEQ ID NO: 141/142, SEQ ID NO: 143/144, SEQ ID NO: 145/146, SEQ ID NO: 147/148, SEQ ID NO: 149/150, SEQ ID NO: 151/152, and SEQ ID NO: 153/154.

Abstract: In certain aspects, provided herein are RNA complexes (e.g., asymmetric RNA complexes, such as asiRNAs or cell penetrating asiRNAs) that inhibit ANGPT2 and/or PDGFB expression and are therefore useful for treating angiogenesis-associated diseases, such as cancer, AMD, and DME.

Abstract: The present invention relates to a composition comprising a repressor of mitofusin gene expression, an inhibitor of mitofusin protein activity, or a mixture thereof as an active ingredient for promoting reprogramming a differentiated cell into a pluripotent stem cell, and a use thereof. The composition according to the present invention increases the efficiency of reprogramming as well as reduces the time required for reprogramming to produce pluripotent stem cells. Therefore, the present composition can be beneficially used to develop the production technology of high efficiency pluripotent stem cell and secure a large-scale culture system. Further, the present composition can be beneficially used to maintain pluripotent stem cells and screen the compounds capable of promoting the reprogramming into pluripotent stem cells.

Abstract: Provided herein are gapmer oligomeric compounds for reduction of target RNA in vivo comprising different nucleotide modifications within one or both wing regions. Also provided are methods of using such oligomeric compounds, including use in animals. In certain embodiments, such compound have desirable potency and toxicity characteristics.

Abstract: Disclosed herein are antisense compounds and methods for decreasing STAT3 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate hyperproliferative diseases.

Abstract: Provided herein is chitosan dually derivatized with arginine and gluconic acid; and methods of making and using the same, e.g., for gene delivery in vivo.

Abstract: The invention relates to novel miRNA markers useful for diagnosis or therapy of disease, in particular for neuronal disorders such as Alzheimer's Disease (AD).

Abstract: Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.

Abstract: This invention relates to interfering RNA (iRNA) molecules and their applications, especially multi-targets iRNA molecules and their applications. The said multi-targets iRNA molecules comprised of a sense strand annealed onto at least one antisense strand, each strand is at least 30 nucleotides in length, the sense or antisense strand has at least two segments, which can target at least two RNAs of different genes, or can target at least two portions of an RNA, and wherein the iRNA does not induce an interferon-response when transfected into a cell. The iRNA molecule can interfere with the translation procedure post-transcription, and the target gene is inhibited or blocked, the iRNA does not induce an interferon-response in vivo. The RNA molecules are the active ingredient in preparation of the drug which can regulate one or many genes function.

Abstract: The present disclosure relates to the finding that microRNA-155 plays a role in inflammation, hematopoiesis and myeloproliferation, and that dysregulation of microRNA-155 expression is associated with particular myeloproliferative disorders. Disclosed herein are methods and compositions for diagnosing an treating disorders, including inflammation and myeloproliferation, modulating the levels of expression of one or more genes selected from the group consisting of Cutl1, Arntl, Picalm, Jarid2, PU.1, Csf1r, HIF1?, Sla, Cepb?, and Bach1, and the like.

Abstract: The invention pertains to amphiphilic dendrimers complexed with Bcl3 siRNA. The amphiphilic dendriplexes described herein are biodegradable and exhibit enhanced siRNA delivery. The amphiphilic dendriplexes containing Bcl3 siRNA inhibit cancer cell/tumor growth in vitro and in vivo without toxicity. Accordingly, an embodiment of the invention provides a method of treating a cancer, particularly, nasopharyngeal carcinoma, by administering to a subject in need thereof, a therapeutic amount of amphiphilic dendrimers complexed with Bcl3 siRNA. Pharmaceutical compositions containing the amphiphilic dendrimers complexed with Bcl3 siRNA are also provided.

Abstract: The present invention relates to a method for producing pools of siRNA molecules suitable for RNA interference.

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. A nucleic acid molecule can have a) a polynucleotide sense strand and a polynucleotide antisense strand; b) each strand of the molecule being from 15 to 30 nucleotides in length; c) a contiguous region of from 15 to 30 nucleotides of the antisense strand being complementary to a sequence of an mRNA encoding p21; and d) at least a portion of the sense strand can be complementary to at least a portion of the antisense strand, and the molecule has a duplex region of from 15 to 30 nucleotides in length.

Abstract: The invention relates to the identification of Secreted Protein, Acidic and Rich in Cysteine (SPARC) as a new therapeutic target in patients with fulminant hepatitis and allows the development of a strategy destined to protect the liver form damage. The invention relates to the treatment of acute liver failure or fulminant hepatitis by administering to a subject in need thereof an agent that inhibits at least partially the expression of SPARC and/or interferes with its biological function.

Abstract: The present invention relates to apparatus, methods, and applications for treating wastewater, and more particularly to biological processes for removing pollutants from wastewater. This invention further relates to apparatus and methods for growing microbes on-site at a wastewater treatment facility, and for economically inoculating sufficient microbes to solve various treatment problems rapidly.

Abstract: The present invention concerns a protein comprising a mutated sequence of SEQ ID No. 1, said mutated sequence of SEQ ID No. 1 comprising at least one mutation A, A?, B, C or C?, in which: mutation A consists of substituting amino acids 43 to 52 of sequence SEQ ID No. 1 with a sequence chosen from DFLAEGLTPR (SEQ ID NO: 159), KATN*ATLSPR (SEQ ID NO: 160) and KATXATLSPR (SEQ ID NO: 161), mutation A? consists of substituting amino acids 47 to 52 of sequence SEQ ID No. 1 with a sequence chosen from TSKLTR (SEQ ID NO: 162), FNDFTR (SEQ ID NO: 163), LSSMTR (SEQ ID NO: 164), PPSLTR (SEQ ID NO: 165) and LSCGQR (SEQ ID NO: 166), mutation B consists of inserting a sequence chosen from DFLAEGLTPR (SEQ ID NO: 159), KATN*ATLSPR (SEQ ID NO: 160), KATXATLSPR (SEQ ID NO: 161), TSKLTR (SEQ ID NO: 162), FNDFTR (SEQ ID NO: 163), LSSMTR (SEQ ID NO: 164), PPSLTR (SEQ ID NO: 165) and LSCGQR (SEQ ID NO: 166), between amino acids 52 and 53 of sequence SEQ ID No.

Abstract: The present disclosure relates to branched and linear chimeric compounds containing both nucleic acid and non-nucleic acid moieties, as well as to polynucleotides. The present disclosure also relates to uses thereof for stimulating an immune response, and to methods for preparation of the branched chimeric compounds.

Abstract: The present invention relates to chemical modification motifs for oligonucleotides. The oligonucleotides of the present invention, such as chemically modified antisense oligonucleotides, can have increased in vivo efficacy. The chemically modified oligonucleotides provide advantages in one or more of potency, efficiency of delivery, target specificity, toxicity, and/or stability. The chemically modified oligonucleotides have a specific chemical modification motif or pattern of locked nucleic acids (LNAs). The oligonucleotide (e.g. antisense oligonucleotide) can target RNA, such as miRNA or mRNA. Also provided herein are compositions comprising the chemically modified oligonucleotides and methods of using the chemically modified oligonucleotides as therapeutics for various disorders, including cardiovascular disorders.