Compositions and methods are provided for the inhibition, treatment and/or prevention of degenerative myelopathy (DM) or amyotrophic lateral sclerosis (ALS).

Compositions and methods are provided for the inhibition, treatment and/or prevention of degenerative myelopathy (DM) or amyotrophic lateral sclerosis (ALS).

The present invention provided an oligonucleotide targeting the core editing-site complementary sequence (ECS) of AZIN1 gene, wherein the core ECS of AZIN1 gene comprises the sequence 5′-GCTTTTCC-3′, and wherein the oligonucleotide comprises one or more nucleotides with sugar modification and one or more modified internucleotide linkages. In another aspect, there is provided a pharmaceutical composition comprising the oligonucleotide as disclosed herein. In another aspect, there is provided a method of inhibiting AZIN1 pre-mRNA editing in a cell, wherein the AZIN1 pre-mRNA editing is mediated by adenosine deaminase acting on RNA-1 (ADAR-1), as well as a method of using the same for the treatment of cancers associated with AZIN1 pre-mRNA editing, including liver cancer.

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.

Provided herein are antisense oligonucleotides (ASOs) specific for Rab13 and Net1, for example specific for a GA-rich region of the 3′-UTR. In some examples, the ASOs are modified. Methods of using these ASOs to reduce the migration of metastatic cancer cells are provided, for example as a use to treat metastatic cancer.

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 present invention aims at establishing a novel therapy for facioscapulohumeral muscular dystrophy.

An oligonucleotide or a pharmaceutically acceptable salt thereof, wherein the oligonucleotide comprises an oligonucleotide of 15-30 bases consisting of a nucleotide sequence complementary to the region of nucleotide Nos. 502-556 or 578-612 of DUX4-fl mRNA consisting of the nucleotide sequence as shown in SEQ ID NO: 1; the 5′ and/or 3′ end of the oligonucleotide may be chemically modified; and the oligonucleotide is capable of switching the splice form of the DUX4 gene from DUX4-fl to DUX4-s. A pharmaceutical drug comprising the above oligonucleotide or a pharmaceutically acceptable salt thereof (e.g. therapeutic for facioscapulohumeral muscular dystrophy).

Active agents that bind to VEGF or a VEGF receptor and reduce the severity of a condition associated with BLB disruption and/or angiogenesis, for example anti-VEGF antibodies or tyrosine kinase inhibitor small molecules, can be locally, regionally or systemically administered to an individual with Meniere's Disease to alleviate symptoms of the disease, for example, due to edema and endolymphatic dysfunction. An effective amount of these compounds can be delivered by intratympanic or intracochlear administration. Other methods of administration include, but are not limited to, topical, parenteral, subcutaneous, intraperitoneal and intranasal. Formulations may be, for example, for immediate release, sustained release, or controlled release.

Methods of treating and preventing diseases associated with fibrosis are disclosed, as well as agents for use in such methods. The methods comprise inhibiting at least one of ITFG1, MFAP4, GRHPR, ABCC4, PAK3, TRNP1, APLN, KIF20A, and† or LTB. In one embodiment, the disease is a liver disease or condition. Also disclosed are methods of promoting regeneration of cells, such as hepatocytes.

The present disclosure provides nucleic acid compositions that incorporate one or more halouracil molecules. More specifically, the present disclosure reveals that the replacement of uracil nucleotides within a microRNA nucleotide sequence with a 5-halouracil increases the ability of the micro-RNA to inhibit cancer progression and tumorigenesis. As such, the present disclosure provides various nucleic acid (e.g., microRNA) compositions having 5-halouracil molecules incorporated in their nucleic acid sequences and methods for using the same. The present disclosure further provides pharmaceutical compositions comprising the modified nucleic acid compositions, and methods for treating cancers using the same.