The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications. In certain embodiments, the present disclosure provides oligomeric compounds having an improved therapeutic index or an increased maximum tolerated dose.

Disclosed is a method of treating a subject who has a neurological disease. In one aspect, the method includes a step of administering an effective dose of a SYF2 antisense or inhibitory nucleic acid to a subject in need thereof, thereby restoring nuclear localization of TDP-43.

The present disclosure provides oligomeric compounds (including oligomeric compounds that are antisense agents or portions thereof) comprising a modified oligonucleotide having at least one modified internucleoside linking group.

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SOD1 gene, as well as methods of inhibiting expression of a SOD1 gene and methods of treating subjects having a SOD1-associated neurodegenerative disease or disorder, e.g., Amyotrophic Lateral Sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), and Down's syndrome (DS), using such dsRNAi agents and compositions.

The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with excess growth hormone using antisense compounds or oligonucleotides targeted to growth hormone receptor (GHR).

Disclosed herein is a method of identifying an L-RNA aptamer specific for a target nucleic acid. According to some embodiments of the present disclosure, the method comprises, in vitro transcribing the DNAs of the DNA library into D-RNAs, followed by identifying target-specific D-RNAs via negative and positive selections, and then producing the L-RNA aptamer based on the identified D-RNA. Also disclosed herein are two aptamers identified by the present method. According to some embodiments of the present disclosure, the two aptamers respectively comprise the nucleotide sequences of SEQ ID NOs: 1 and 2.

This invention encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting Hepatitis B virus in a subject. The compounds have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers, and the compounds are targeted to a sequence of an HBV genome.

Disclosed is an oligonucleotide-modified nucleic acid containing at least one 1-β-D-arabinofuranosylcytosine as a modified nucleic acid having therapeutic efficacies and guanosine. More particularly, a novel oligonucleotide-modified nucleic acid containing at least one modified nucleic acid (N) having therapeutic efficacies and being rich in guanosine (G) is synthesized and the fact that the novel oligonucleotide-modified nucleic acid has excellent apoptotic activities on blood cancer cells and drug-resistant blood cancer cells is identified. Based on this, provided is a composition for preventing, ameliorating or treating blood cancer, containing the novel oligonucleotide-modified nucleic acid, and the novel oligonucleotide-modified nucleic acid or a pharmaceutically acceptable salt thereof as an active ingredient.

The invention provides xeno-nucleic acid particle display libraries, methods for identifying functional non-natural nucleic acid (XNA) aptamers using the particle display libraries, and compositions comprising XNA aptamers identified by screening candidate molecules using the xeno-nucleic acid particle display libraries.

The invention provides for a method for selectively reducing the expression of a mutant mRNA and/or protein having an expanded nucleotide repeat relative to a wild-type mRNA, comprising contacting a cell with an antisense oligonucleotide of sufficient length and complementarity to the expanded nucleotide repeat. More particularly it relates to selectively reducing the expression of mutant Huntington protein associated with Huntington's disease. The antisense oligonucleotide comprising either a nucleotide or a repeated three nucleotide sequence as defined in the claims.