Abstract: Aspects of the disclosure provide compounds, compositions, and methods for modulating the expression or activity of plasma prekallikrein (PKK). In some aspects, the compounds, compositions, and methods of the disclosure can be used to reduce the expression of PKK mRNA in a cell or animal. In some aspects, the compounds, compositions, and methods of the disclosure can be used to reduce the expression of PKK protein in a cell or animal.

Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting IRF4 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with IRF4.

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: The present invention relates to a double-stranded oligonucleotide which can highly specifically and efficiently inhibit an amphiregulin expression and, preferably, a double-stranded oligonucleotide comprising a sequence in the form of RNA/RNA, DNA/DNA or DNA/RNA hybrid, a double-stranded oligonucleotide structure comprising the double-stranded oligonucleotide, nanoparticles comprising the double-stranded oligonucleotide structure, and a fibrosis or respiratory disease preventive or therapeutic use thereof.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result.

Abstract: microRNAs and compositions comprising same for the treatment and diagnosis of serotonin-, adrenalin-, noradrenalin-, glutamate-, and corticotropin-releasing hormone-associated medical conditions are provided.

Abstract: A method for reducing or substantially preventing soft tissue calcification is provided. The method includes administering at least one of a downregulator of at least one plasmin inhibitors and plasmin(ogen) to a subject in need thereof, wherein the at least one plasmin inhibitor includes alpha2-antiplasmin. Further disclosed is a method comprises administering the compound subsequent to muscle injury, and the method further comprising administering an antifibrinolytic.

Abstract: The invention is directed to methods and compositions for collecting a non-placental biological samples of cells and quantifying and comparing levels of expression of microRNAs to characterize a preeclampsia-related condition. The samples may be collected before or after an intervention or may be collected over a period of time. One of the samples may be a control sample. Patients may then be treated according to their response.

Abstract: The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with target gene expression or inhibit target gene expression and therapeutic uses of such products.

Abstract: RNA from a biological fluid is stabilized during isolation and/or storage using DNA. In especially preferred aspects, the RNA is cfRNA and/or ctRNA, and the biological fluid is blood.

Abstract: The present invention provides a novel antisense oligonucleotide and a composition for preventing or treating glycogen storage disease type Ia. The present invention provides an antisense oligonucleotide which hybridizes with a pre-mRNA sequence derived from a region including at least one of a base at position 42911000, a base at position 42911004, and a base at position 42911005 in a base sequence of human chromosome 17 of GRCh38/hg38 and has activity to inhibit aberrant splicing of pre-mRNA of c.648G>T variant G6PC.

Abstract: The present invention provides cells which have a high ability to propagate influenza virus, are suitable for use in production of an influenza virus for preparing a vaccine, and are able to be cultured in vitro, and a method for producing an influenza virus using the cells. That is, the present invention provides cells for producing an influenza virus in which expression of one or more genes that encode proteins involved in an effect of suppressing influenza virus production in a cell is suppressed and the gene is at least one selected from the group including ACTG1 gene and the like, and a method for producing an influenza virus that includes infecting the cells for producing an influenza virus with an influenza virus and then culturing.

Abstract: The present disclosure relates to a composition for preventing or treating fibrosis including an inhibitor of cyclin-dependent kinase 17 (CDK17) expression or activity. According to the present disclosure, the inhibitor of CDK17 expression or activity significantly reduces collagen production and cell activity and viability in activated hepatic stellate cells (liver fibrosis cell model), renal tubular epithelial cells (renal fibrosis cell model) in which fibrosis is induced by TGF-? treatment, and alveolar epithelial cells (lung fibrosis cell model) in which fibrosis is induced by TGF-? treatment, indicating that the composition of the present disclosure has an excellent effect in preventing or treating fibrosis.

Abstract: Oligonucleotides are provided herein that inhibit TMPRSS6 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with hepcidin deficiency or suppression.

Abstract: The present invention pertains to a method for the identification of genetic variants that are associated with the severity of an infectious disease. The invention further pertains to a set of genetic factors associated with the severity of Human respiratory syncytial virus (HRSV) infection, for example in human infants. The genetic polymorphisms identified according to the present invention are for use in the diagnostic of infectious diseases and patient stratification in order to avoid or reduce the occurrence of fatal events during infection or to elect the most appropriate therapeutic approach to treat the disease.

Abstract: A nanoparticle delivery system designed for sustained delivery of microRNA-150 (miR-150) to FLT3-overexpressing acute myeloid leukemia (AML) cells, the delivery system comprising poly(amidoamine) (PAMAM) dendrimers complexed with miR-150, wherein at least one dendrimer is surface-functionalized with a ligand specific for FLT3 receptor, and methods for treating AML characterized by FLT3-overexpression are provided.

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of sodium voltage-gated channel alpha subunit 2 (SCN2A) in a subject. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a sodium voltage gated channel alpha subunit 1 (SCN1A) related disease or disorder (e.g., Dravet syndrome) in a subject in need.

Abstract: A nucleotide sequence is shown in SEQ ID NO.1. The gene encodes mannosyltransferase I. The gene plays an important role in the normal reproductive development of the Nilaparvata lugens (Stål). Inhibition of the function of the gene may reduce the survival rate of the Nilaparvata lugens (Stål) and hinder embryonic development. With respect to reduction of the survival rate of the Nilaparvata lugens (Stål) and hindering of embryonic development, the present invention can reduce the harm of the Nilaparvata lugens (Stål) to rice by killing the Nilaparvata lugens (Stål). By using the characteristic that the nucleotide sequence of a highly conserved target gene has no homology with the nucleotide sequence of natural enemies of the Nilaparvata lugens (Stål), RNA interference is performed at a nucleic acid level, to avoid the harm to non-target organisms such as natural enemies, thereby realizing green control of the Nilaparvata lugens (Stål) while controlling pests.

Abstract: Antisense oligonucleotides target the mutation in intron 26 of the CEP290 gene and reduce inclusion of the aberrant exon into the CEP290 mRNA. The oligonucleotides include no more than 3 consecutive guanosines, have no more than 60% guanosine nucleobases, include at most one CpG sequence, and/or do not have the potential to form a hairpin comprising 3 or more consecutive complementary base pairs.

Abstract: This invention relates to compounds, compositions, and methods useful for reducing ?-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.