An oligonucleotide variant according to an embodiment of the present disclosure has a structure of Formula 1 may exhibit excellent in-vivo stability and anticancer effects:

(N).sub.x-[TGG].sub.m[TTG][TGG].sub.n-(M).sub.y  [Formula 1] wherein, N and M are independently deoxyuridine (dU), deoxycytidine (dC), uridine (U), or cytidine (C), in which a halogen or hydroxy group is bound to 5- or 2′-position thereof; x and y are independently integers of 0 to 10 (except when x and y are simultaneously 0), n is an integer of 1 to 10; and m is an integer of 1 to 10.

The present disclosure describes chemically-stabilized RNA substrates that hybridize to poly-A binding protein (PABP) with high specificity in vitro, as well as their use in impairs nascent translation in a PABP-dependent mechanism in cells, thereby treating pain.

The invention relates to the treatment of genetic dilated cardiomyopathies using expressible modulators of the Wnt pathway or TGF-β pathway, preferably using gene transfer.

Provided herein are, inter alia, nucleic acid compounds useful for targeting CTLA-4-expressing cells and modulating cell activity of the CTLA-4-expressing cells. The compositions provided herein may be part of pharmaceutical compositions and may be used for treatment of cancer, inflammatory diseases, infectious diseases or metabolic diseases.

The present invention relates to the combination of a Dbait molecule with a protein kinase inhibitor for treating cancer.

Various embodiments provide STOPS™ polymers that are S-antigen transport inhibiting oligonucleotide polymers, processes for making them and methods of using them to treat diseases and conditions. In some embodiments the STOPS™ modified oligonucleotides include an at least partially phosphorothioated sequence of alternating A and C units having modifications as described herein. The sequence independent antiviral activity against hepatitis B of embodiments of STOPS™ modified oligonucleotides, as determined by HBsAg Secretion Assay, is greater than that of a reference compound.

The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.

Disclosed herein, inter alia, are oligonucleotide inhibitors of the Nuclear Factor Kappa-Light-Chain-Enhancer of Activated B Cells (NF-κB) signaling pathway and methods of use thereof.

Various embodiments provide STOPS™ polymers that are S-antigen transport inhibiting oligonucleotide polymers, processes for making them and methods of using them to treat diseases and conditions. In some embodiments the STOPS™ modified oligonucleotides include an at least partially phosphorothioated sequence of alternating A and C units having modifications as described herein. The sequence independent antiviral activity against hepatitis B of embodiments of STOPS™ modified oligonucleotides, as determined by HBsAg Secretion Assay, is greater than that of a reference compound.

Provided herein are, inter alia, nucleic acid compounds useful for targeting CTLA-4-expressing cells and modulating cell activity of the CTLA-4-expressing cells. The compositions provided herein may be part of pharmaceutical compositions and may be used for treatment of cancer, inflammatory diseases, infectious diseases or metabolic diseases.