Abstract: The present invention relates to the combination of a PARP inhibitor with a Dbait molecule for treating cancer.

Abstract: The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.

Abstract: The present invention provides an application of lncRNAs ENST00000607393 siRNA in preparing a preparation for treating glaucoma. The present invention clarifies the correlation between the expression level of ENST00000607393 and the calcification of human primary trabecular meshwork cells. The expression level of ENST00000607393 in human primary trabecular meshwork cells is firstly down-regulated to 46.21% of a control group by siRNA interference. Under the condition of intervening with the cells by a 500 ?mol/L hydrogen peroxide solution for 48 hours, it is observed that compared with the control group, the down-regulation of ENST00000607393 expression can significantly decrease the ALP activity in human primary trabecular meshwork cells, and thus significantly decrease the calcification level of human primary trabecular meshwork cells to play a role of treating or preventing glaucoma.

Abstract: Provided herein are methods for using RNAi molecules targeting a proteasome beta 5 (PSMB5) gene for controlling Coleopteran insects, methods for producing RNAi molecules targeting PSMB5, and compositions comprising RNAi molecules targeting PSMB5.

Abstract: The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the ketohexokinase (KHK) gene, and methods of using such RNAi agents to inhibit expression of KHK and methods of treating subjects having a KHK-associated disorder, e.g., liver disease (e.g., fatty liver, steatohepatitis), dyslipidemia (e.g., hyperlipidemia, high LDL cholesterol, low HDL cholesterol, hypertriglyceridemia, postprandial hypertriglyceridemia), disorders of glycemic control (e.g., insulin resistance, diabetes), cardiovascular disease (e.g., hypertension, endothelial cell dysfunction), kidney disease (e.g., acute kidney disorder, tubular dysfunction, proinflammatory changes to the proximal tubules), metabolic syndrome, adipocyte dysfunction, visceral adipose deposition, obesity, hyperuricemia, gout, eating disorders, and excessive sugar craving.

Abstract: The present invention provides for methods of delivering polynucleotides to subterranean plant pests. In particular, the present invention relates to methods of reducing the binding of a composition, comprising polynucleotide and a cationic polymer, to soil by substantially quenching positively charged residues with a quenching agent. The invention also comprises compositions comprising a cationic polymer, a polynucleotide and a quenching agent.

Abstract: The present invention relates to a double-stranded oligo RNA structure comprising double-stranded miRNA, and a composition for preventing or treating cancer, containing the same. More specifically, the present invention relates to an anti-cancer pharmaceutical composition, containing: a double-stranded oligo RNA structure comprising miR-3670, miR-4477a and miR-8078, and characterized by a method for effectively inhibiting cancer cell proliferation or inducing cancer cell apoptosis; and a pharmaceutically acceptable carrier.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) targeting a PROC gene, and methods of using the dsRNA to inhibit expression of PROC.

Abstract: Disclosed herein are antisense compounds and methods for decreasing Ataxin 2 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotropic sclerosis (ALS), and parkinsonism.

Abstract: The present invention discloses c-MYC-siRNA formulation as a potential therapeutic target for cisplatin-resistant ovarian cancer. It is disclosed targeting c-MYC with small interfering RNA (siRNA) in the cisplatin-resistant ovarian cancer cell line inducing a significant cell growth arrest and inhibition of cell proliferation. Apoptosis and arrest of cell cycle progression were also observed after c-MYC-siRNA-based silencing of c-MYC. Furthermore, delivering nanoliposomal c-MYC-siRNA, decreased tumor weight and number of tumor nodules compared with a liposomal-negative control siRNA.

Abstract: The present invention relates to compositions and methods for siRNA therapeutics for prevention and treatment of Middle East Respiratory Syndrome Corona Virus (MERS-CoV) infections. The compositions include a pharmaceutical composition comprising siRNA cocktails that target viral genes and pharmaceutically acceptable polymeric nanoparticle carriers and liposomal nanoparticle carriers.

Abstract: Described herein are methods of expanding a population of hematopoietic stem cells by contacting the population of hematopoietic stem cells with an effective amount of an inhibitor of G-protein coupled receptor 65 (GPR65) and providing a population of expanded, substantially undifferentiated hematopoietic stem cells. Exemplary GPR65 inhibitors include siRNAs and certain sphingolipids. Also described are populations of expanded hematopoietic stem cells produced by the methods, media and kits containing GPR65 inhibitors, and methods for administering an expanded population of hematopoietic stem cells to patients.

Abstract: There is provided a medical agent for use in a method of treatment of nonalcoholic fatty liver disease (NAFLD) or hepatocellular carcinoma (HCC), wherein the medical agent comprises a polynucleotide and is capable of silencing or knocking out the PKLR gene.

Abstract: The present invention provides a suppression type antisense oligonucleotide targeting TDP-43 mRNA, containing a nucleotide sequence complementary to a sequence consisting of at least 10 continuous nucleotides in a nucleotide sequence shown in any of SEQ ID NOs: 2-4, and a promotion type antisense oligonucleotide targeting TDP-43 mRNA, containing a nucleotide sequence complementary to a sequence consisting of at least 10 continuous nucleotides in a nucleotide sequence shown in SEQ ID NO: 5.

Abstract: The present invention discloses the use of miR-4714, miR-4521, and miR-1292 as diagnostic markers for osteosarcoma. High-throughput sequencing and QPCR experiments prove that the levels of miR-4714, miR-4521, and miR-1292 in the blood of normal and osteosarcoma patients are significantly different, and therefore miR-4714, miR-4521, and miR-1292 can be used as a molecular marker for the diagnosis of osteosarcoma for developing products for the diagnosis of osteosarcoma. The diagnostic product has the following advantages: non-invasive, rapid, sensitive, and has broad application prospects in clinical practice.

Abstract: The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject. The present embodiments provided herein are directed to compounds and compositions useful for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP). In certain embodiments, P23H rhodopsin inhibitors provided herein are allele-specific antisense compounds targeted to a P23H mutant allele that are capable of selectively inhibiting expression of P23H rhodopsin mutant protein to a greater extent than wild-type protein.

Abstract: Compounds and compositions for the transient expression of exogenous telomerase activity in a cell are provided. The compounds and compositions, which relate to a ribonucleic acid coding for a telomerase reverse transcriptase, are useful in the extension of telomeres in cells needing such treatment. Such cells include, for example, cells that contain shortened telomeres and cells from subjects that may benefit from telomere extension, for example subjects that suffer from, or are at risk of suffering from, age-related or other illnesses. Also provided are methods of extending telomeres through the administration of the provided compounds and compositions to animal cells, either in vitro or in vivo, and kits including the compounds or compositions and instructions for use.

Abstract: Aspects of the invention relate to methods for treating ALS comprising administering to a subject in need thereof a therapeutically effective amount of a nucleic acid molecule that is directed against a gene encoding superoxide dismutase 1 (SOD1).

Abstract: Methods and compositions for increasing the suppressive function of regulatory T-cells (Tregs) are provided.

Abstract: Disclosed herein are pharmaceutical compositions and methods for inhibiting oxidative stress in a subject having atrial or ventricular arrhythmias, ventricular failure or heart failure. The methods include administering an effective amount of a NOX2 inhibitor agent to the subject, wherein said administering is under conditions such that a level of oxidative stress in myocardial tissue is reduced or eliminated. The pharmaceutical compositions include a NOX2 inhibitor agent.