Abstract: The present invention relates to antisense oligonucleotides that are capable of modulating expression of ATXN2 in a target cell. The oligonucleotides hybridize to ATXN2 mRNA. The present invention further relates to conjugates of the oligonucleotide and pharmaceutical compositions and methods for treatment of neurodegenerative diseases such as spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), Alzheimer's frontotemporal dementia (FTD), parkinsonism and conditions with TDP-43 proteinopathies using the oligonucleotide.

Abstract: The present invention relates to inhibitors of gangliosides metabolism for treating motor neuron diseases, in particular hereditary spastic paraplegias.

Abstract: Disclosed herein are methods involving the targeting of 5HT biosynthesis in gut insulin-negative cells to convert them into insulin-positive cells. Also, disclosed are methods for treating a disease or disorder in a mammal, preferably a human, associated with impaired pancreatic endocrine function, by administering a therapeutically effective amount of an enumerated active agent that reduces the expression, biosynthesis, signaling or biological activity of serotonin or increases its degradation, wherein administering comprises delivering the agent to Gut Ins? cells in the mammal. Other embodiments of the method are directed to therapy wherein an agent that significantly reduces FOXO1 expression, biosynthesis, signaling or biological activity or increases its degradation is administered in addition to the agent that reduces serotonin, or alternatively an agent that reduces FOXO1 expression is targeted to serotonin-positive gut enteroendocrine cells.

Abstract: Virus vectors wherein each of the virus vectors expresses a sequence targeting a consensus conserved nucleic acid sequence, which when expressed in cells, functions as a modulator for nucleic acid encoding a domain.

Abstract: A method of treating a subject suffering from a neurodegenerative disease characterized by insufficient autophagy is provided, the method comprising administering to the subject an effective amount of a composition that inhibits N-deacetylase N-sulfotransferase (NDST). Further provided is a method of identifying a modulator of autophagy.

Abstract: A method for producing RNA is provided. Objective RNA is produced by culturing a coryneform bacterium having an expression unit for the objective RNA, which has been modified so that the activity of ribonuclease III is reduced, in a medium, to express the objective RNA and accumulate the objective RNA in cells of the bacterium, and collecting the objective RNA from the cells.

Abstract: Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.

Abstract: Provided are polynucleotides that find use, e.g., for detecting RNase activity. In certain embodiments, the polynucleotide comprises a tRNA operably linked to a pre-microRNA (pre-miRNA), and an aptamer that binds to a detectable label. According to some embodiments, the detectable label is a fluorescent dye, non-limiting examples of which include malachite green, tetramethylrosamine, sulforhodamine B, and a triphenylmethane dye. Also provided are compositions that include the polynucleotides of the present disclosure. Also provided are kits that include a polynucleotide of the present disclosure and a detectable label to which the aptamer of the polynucleotide binds.

Abstract: The purpose of the present invention is to provide novel miR-143 derivatives described herein that can be used as oligonucleotide therapeutics.

Abstract: The invention provides methods of treating polyglutamine diseases, e.g., spinocerebellar ataxia Type 6, in a subject, comprising administering to the subject an IRES inhibitor in an amount effective for treating the SCA6 in the subject. Also provided herein are the IRES inhibitors, and pharmaceutical compositions comprising the same.

Abstract: The present application discloses nucleic acid aptamer based signaling polynucleotides (SPNs) that specifically bind an allergen protein. Provided in the present invention include aptamers, SPNs, SPN-complement complexes, magnetic particle conjugates, DNA printed glass slides and detection agents, and detection methods using the same for detecting the presence, or absence of an allergen protein in a food sample.

Abstract: The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.

Abstract: Oligomers can be prepared from bicyclic nucleosides. The nucleosides can be a compound of formula (I) in which each of T1 and T2 is independently OR1 or OR2; R1 is H or a hydroxyl protecting group, R2 is a phosphorus moiety; and Bx is a nucleobase. The compounds, bicyclic nucleosides and oligomers are useful for the prevention, treatment or diagnosis of muscular dystrophy.

Abstract: Provided are antisense morpholino oligomers targeted against bacterial virulence factors such as genes that contribute to antibiotic resistance or biofilm formation, or genes associated with fatty acid biosynthesis, and related compositions and methods of using the oligomers and compositions, for instance, in the treatment of an infected mammalian subject.

Abstract: Provided are constructs and methods for RNA promoter identification.

Abstract: The present invention is drawn to nucleic acid aptamer based signaling polynucleotides (SPNs) for allergen detection in samples. Disclosed herein include compositions, compounds, assays and methods of using said SPNs to detect one or more allergens in a sample, particularly food allergens in a food product.

Abstract: The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with gene expression or inhibits its expression and therapeutic uses such as for the treatment of disease and disorders.

Abstract: The present invention provides methods for treating Parkinson's Disease (PD), e.g., PD associated with a genetic mutation in a glucocerebrosidase (GBA) gene or a leucine rich repeat kinase 2 (LRRK2) gene. The methods comprise administering to the subject a modulator, e.g., an inhibitor, of p53-inducible gene 3 (PIG3).

Abstract: The invention relates to methods of improving efficacy and enhancing silencing of target genes using Ago2 protein-dsRNA complex (RNP) of the invention.

Abstract: Aspects of the present invention include methods and compositions related to the modulation of molecules regulating the regenerative potential of cells and tissues in the embryonic state and the loss thereof in later fetal and adult stages of development. Said methods and compositions have uses in research in stem cell biology and in increasing regenerative potential in fetal and adult tissues otherwise incapable of regeneration.