C12N2310/3231

TREATMENT AND PREVENTION OF THROMBOSIS USING AN ANTI-MIR

Disclosed are anti-miR compositions and methods of use of the same for treatment of or reducing the occurrence of thrombosis and thrombosis related diseases and disorders by reducing platelet activation.

EGFR NUCLEIC ACIDS AND USES THEREOF
20170362597 · 2017-12-21 ·

Disclosed herein are molecules and pharmaceutical compositions that mediate RNA interference against EGFR. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that mediate RNA interference against EGFR.

ANTISENSE OLIGONUCLEOTIDES TARGETING ALPHA-SYNUCLEIN AND USES THEREOF

The present disclosure relates to antisense oligonucleotides, which target SNCA mRNA (e.g., at an intron exon junction) in a cell, leading to reduced expression of SNCA protein. Reduction of SNCA protein expression is beneficial for the treatment of certain medical disorders, e.g., a neurological disorder.

USE OF SBDS INHIBITORS FOR TREATING HEPATITIS B VIRUS INFECTION

The present invention relates to a SBDS inhibitor for use in treatment of an HBV infection, in particular a chronic HBV infection. The invention in particular relates to the use of SBDS inhibitors for destabilizing cccDNA, such as HBV cccDNA. The invention also relates to nucleic acid molecules which are complementary to SBDS and capable of reducing the level of a SBDS mRNA. Also comprised in the present invention is a pharmaceutical composition and its use in the treatment of a HBV infection.

Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals

The present invention provides compounds and methods for modulating target nucleic acids found in organelles or sub-organelles of cells. The invention includes, but is not limited to compounds and methods that modulate target nucleic acids in a sub-nuclear organelle, such as the nucleolus and/or a cajal body. In certain embodiments, the cell is in an animal.

OLIGONUCLEOTIDES FOR SPLICE MODULATION OF CARD9

The present invention relates to splice modulating oligonucleotides (oligomers) that are complementary to the CARD9 pre-mRNA, for inhibiting the inclusion of a CARD9 exon 11 in CARD9 mRNA. The oligonucleotides of the invention are useful in the treatment of inflammatory diseases such as IBD.

EXTRACELLULAR VESICLES WITH STAT3-ANTISENSE OLIGONUCLEOTIDES

The present disclosure relates to modified extracellular vesicles, e.g., exosomes, comprising an antisense oligonucleotide (ASO), which is capable of reducing and/or inhibiting expression of STAT3 mRNA and/or STAT3 protein. ASOs that can be used with the modified extracellular vesicles are also disclosed. Also provided herein are methods for using the exosomes and ASOs to treat and/or prevent diseases, such as cancer.

CYTOTOXIC T-LYMPHOCYTE BINDING APTAMERS

Provided herein are aptamers that target cytotoxic T-lymphocyte and methods of use thereof.

BISPECIFIC PERSONALIZED APTAMERS

Provided herein are bispecific personalized aptamers that induce the cell death of cancer cells and methods of use thereof.

Nucleic acid molecules capable of modulating target gene expression and uses thereof

This application pertains to a hairpin nucleic acid molecule capable of modulating expression of a target gene and a use thereof. A nucleic acid molecule according to an embodiment can modulate expression of a target gene in a specific manner for cells in which a miRNA hybridizable therewith is present, finding advantageous applications in compositions for regulating expression of a target gene or pharmaceutical compositions for treating diseases.