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: 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: 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: 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.

Abstract: The present invention relates to use of miR-18b for prevention, treatment, or diagnosis of muscle diseases or neuromuscular diseases, and specifically it was confirmed that, in a muscle disease by gene mutations model, gene mutations reduce miR-18b expression, cause dysregulation of miR-18b signaling pathways, and thus induce calcium signaling, cell differentiation inhibition, and apoptosis. Therefore, miR-18b of the present invention may be used as a target factor for diagnosing and treating muscle diseases caused by gene mutations such as ALS and DMD.

Abstract: The invention relates to the application of the ERH gene in the preparation of bladder cancer diagnosis and treatment products. The ERH gene is found to have a good correlation with bladder cancer. In addition, an RNA interference vector is constructed to perform cell function experiments to study the effect of ERH gene on the proliferation and clonality of bladder cancer cells. The present invention provides a research basis for clinical diagnosis and treatment of bladder cancer and has a good application prospect.

Abstract: The invention provides a medication and a diagnostic kit for inhibiting metastasis and invasion of breast cancer, an shRNA molecule for silencing expression of human LINC01614 and applications thereof. The shRNAs obtained by the invention can interfere with the expression of LINC01614, thereby reducing the migration and invasion ability of tumor cells, inhibiting the expression of EMT proteins, and inhibiting tumor formation and lung metastasis in an animal model in vivo. The invention provides a new solution for targeted therapy of breast cancer. Therefore, the kit for diagnosing metastasis and invasion of breast cancer and the medication for treating metastasis and invasion of breast cancer are developed. The invention provides a new way and strategy for diagnosing and treating metastasis and invasion of breast cancer.

Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include Parkinson's disease.

Abstract: Aspects of the invention relate to antisense oligonucleotides directed to the interleukin 17 receptor (IL-17R), and other targets. Spherical nucleic acid formulations or compositions of antisense oligonucleotides and related methods of treatment are also provided.

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.

Abstract: Provided are aptamer compositions and their use for treating cancer.

Abstract: The present disclosure includes cationic carrier units comprising (i) a water soluble polymer, (ii) a positively charged carrier, and (iii) an adjuvant moiety, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.

Abstract: Provided are compounds, methods, and pharmaceutical compositions for increasing the amount or activity of STMN2 RNA in a cell or animal, and in certain embodiments increasing the amount of STMN2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include ataxia, neuropathy, synaptic dysfunction, deficits in cognition, and decreased longevity. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), and dementia with Lewy bodies (DLB).

Abstract: The present disclosure, at least in part, relates to an engineered RNA (e.g., microRNA and sgRNA), in the absence of an input signal, that is engineered to have a large enough energy gap between the formations of a first secondary structure, which is unrecognizable by an actuator, and a second secondary structure, which is recognizable by an actuator (e.g., Drosha and Cas protein).