This disclosure relates to novel huntingtin targets. Novel oligonucleotides for the treatment of Huntington's disease are also provided.

Provided herein are branched oligonucleotides exhibiting efficient and specific tissue distribution, cellular uptake, minimum immune response and off-target effects, without formulation.

The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.

The presently-disclosed subject matter relates to an artificial RNA nanostructure and method of use thereof. In particular, the presently-disclosed subject matter relates to RNA nanoparticles and RNA dendrimers, and methods of disease diagnosis and treatments using the RNA nanostructure and RNA dendrimers.

A method is provided for generating RNA nanoparticles having modified nucleotides and/or having increased nuclease resistance where the RNA nanoparticles are formed cotranscriptionally by T7 RNA polymerase in the presence of manganese ions.

Provided herein are methods, compounds, and compositions for reducing expression of transthyretin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate transthyretin amyloidosis, or a symptom thereof.

The present document describes nucleic acid structures comprising a plurality of annealed motifs that are made from complementary oligonucleotides having domains with sequences complementary to other nucleotides of the motif. The annealed motifs may be anchored to surfaces, and functional elements may be attached to the annealed motifs. The nucleic acid structures may used to make sensors therefrom. The present document also describes methods to generate said nucleic acid structures.

The present invention relates to a highly efficient aptamer complex comprising a branched DNA and an aptamer, and a pharmaceutical use thereof. More specifically, the aptamer complex of the present invention relates to a highly efficient aptamer complex including a Y-shaped DNA as the branched DNA and using vascular endothelial growth factor (VEGF) as a target molecule. The aptamer complex of the present invention and a pharmaceutical composition comprising the same as an active ingredient are expected to be widely used in the medical field since the binding efficiency with the target molecule is more remarkable than that of the conventional aptamer.

The presently-disclosed subject matter relates to an artificial RNA nanostructure molecule and method to treat brain tumor in a subject. More particularly, the presently disclosed subject matter relates to a RNA nanostructure containing a multiple branched RNA nanoparticle, a brain tumor targeting module, and an effective amount of a therapeutic agent. Further, the presently disclosed subject matter relates to a method of using the RNA nanostructure composition to treat brain tumor in a subject having or at risk of having brain tumor.

This disclosure relates to novel huntingtin targets. Novel oligonucleotides for the treatment of Huntington's disease are also provided.