Disclosed herein are antisense oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA at the 5′-end of UBE3A-AS, which is downstream of SNORD115-45 snoRNA. Also disclosed are pharmaceutical compositions and methods for treatment of Angelman syndrome.

The present application relates to the field of inflammatory diseases, particularly to inflammatory diseases characterized by an M1 macrophage response, even more particularly to sepsis and to Trypanosoma infection. The invention provides substances modulating miR210 expression and/or activity, in particular RNA molecules inhibiting miR210 expression and/or activity and medical uses of these miR210 inhibitors. Methods are disclosed to screen for medicaments for treating sepsis.

The present invention provides an antisense oligomer having the base sequence depicted in SEQ ID NO: 26, an antisense oligomer having a base sequence resulting from substitution, deletion, insertion, or addition of 1 to 6 bases in the base sequence depicted in SEQ ID NO: 26, a pharmaceutically acceptable salt thereof, or a pharmaceutically acceptable hydrate thereof, an oligonucleotide conjugate in which the antisense oligomer is bound with a molecule capable of binding to an asialoglycoprotein receptor, and a pharmaceutical composition containing the same.

The present invention relates to antisense oligonucleotides that are capable of reducing expression of PD-L1 in a target cell. The oligonucleotides hybridize to PD-L1 mRNA. The present invention further relates to conjugates of the oligonucleotide and pharmaceutical compositions and methods for treatment of viral liver infections such as HBV, HCV and HDV; parasite infections such as malaria, toxoplasmosis, leishmaniasis and trypanosomiasis or liver cancer or metastases in the liver using the oligonucleotide.

Provided are oligomeric compounds, methods, and pharmaceutical compositions for DMPK the amount or activity of DMPK RNA in a cell or animal, and in certain instances reducing the amount of DMPK protein in a cell or animal. Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat type 1 myotonic dystrophy.

The disclosure includes antisense oligonucleotides, including gapmer antisense oligonucleotides, along with methods of making and use, e.g., against HBV.

The disclosure includes antisense oligonucleotides, including gapmer antisense oligonucleotides, along with methods of making and use, e.g., against HBV.

Provided are a nucleic acid with improved ability to inhibit the expression of the MEX3B gene with low occurrence of cytotoxicity side effects, an MEX3B gene expression inhibiting agent that includes the nucleic acid, a method for inhibiting MEX3B gene expression, and a prophylactic or therapeutic drug for a disease caused by MEX3B gene expression. The present invention is a nucleic acid that is any of (1) to (3): (1) an oligonucleotide that comprises the nucleotide sequence represented by SEQ ID NO: 1 or 2; (2) an antisense oligonucleotide that inhibits expression of the MEX3B gene and that comprises a nucleotide sequence derived by deletion, substitution, and/or addition of one or two nucleotides in the nucleotide sequence represented by SEQ ID NO: 1 or 2; and (3) an antisense oligonucleotide that inhibits expression of the MEX3B gene and that includes the nucleotide sequence represented by SEQ ID NO: 3.

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 herein are conditional siRNAs activatable by pro-hypertrophic RNA sequences and use thereof for treating conditions such as cardiac hypertrophy. The conditional siRNAs target calcineurin or HDAC2.