Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of APP RNA in a cell or animal, and in certain instances reducing the amount of APP 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 cognitive impairment, including a decline in memory and language skills, behavioral and psychological symptoms such as apathy and lack of motivation, gait disturbances and seizures, progressive dementia, and abnormal amyloid deposits.

Provided herein are methods for the treatment of polycystic kidney disease, including autosomal dominant polycystic kidney disease, using modified oligonucleotides targeted to miR-17.

Disclosed are a 5′-modified nucleoside and a nucleotide using the same. The nucleoside of the present invention is represented by the formula (I) below. The 5′-modified nucleoside of the present invention is usable as a substitute for a phosphorothioate-modified nucleic acid, which has a risk of, for example, accumulation in a specific organ. The 5′-modified nucleoside also has excellent industrial productivity because a diastereomer separation step is not involved in the production process thereof.

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The object of the present invention is to provide a nucleic acid agent that is efficiently delivered to the nervous system, for example, the central nervous system to which drug delivery can be prevented by BBB, and produces an antisense effect on the target transcriptional product at the delivery site, and a composition comprising the same. In an embodiment, the present invention provides a double-stranded nucleic acid complex formed by annealing a first nucleic acid strand that hybridizes to a part of a target transcriptional product and has an antisense effect on the target transcriptional product, and a second nucleic acid strand that comprises a base sequence complementary to the first nucleic acid strand and is bound to a C.sub.22-35 alkyl group optionally substituted with a hydroxy group or an analog thereof.

The present invention relates to an oligomer conjugate for use in the treatment of a viral disorder. The oligomer conjugate comprises: a) an oligomer capable of modulating a target sequence in HBx and/or HBsAg of Hepatitis B Virus (HBV) to treat said viral disorder; and b) a carrier component capable of delivering the oligomer to the liver which is linked, preferably conjugated, to the oligomer.

Disclosed herein are antisense compounds and methods for decreasing Tau mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Tau-associated diseases, disorders, and conditions.

The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.

The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to ATXN3 pre-mRNA sequences, which are capable of inhibiting the expression of ATXN3 protein. Inhibition of ATXN3 expression is beneficial for the treatment of spinocerebellar ataxia

The present disclosure relates to methods for the inhibition of proteins involved in the assembly and or secretion of HBV SVP by inhibiting the activity of casein kinase 1 isoform delta, DNAJB12, and/or microtubule-actin crosslinking factor 1.

The invention relates generally to tRNA-based effector molecules having a non-naturally occurring modification and methods relating thereto.