Abstract: The present disclosure relates to miRNA expression constructs, such as for expression of multiple miRNAs and use thereof to knockdown target gene expression. In some aspects, the expression constructs include a promoter element, a spacer sequence and a miRNA coding sequence. In some aspects, constructs provide enhanced immune cell function.

Abstract: The invention relates to antisense oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA in a eukaryotic cell, wherein said oligonucleotide does not, in itself, form an intramolecular hairpin or stem-loop structure, and wherein said oligonucleotide comprises a cytidine (a non-complementary nucleotide) or a uridine in a position opposite to the target adenosine to be edited in the target RNA region.

Abstract: According to a composition, kit, and method for diagnosing resistance to a taxane anticancer agent, a composition for treating taxane cancer agent-resistant cancers, and a method for screening materials for the same, taxane anticancer agent-resistant cancers may be efficiently diagnosed or treated, and candidate materials that may treat taxane anticancer agent-resistant cancers may be efficiently screened.

Abstract: Provided herein are compositions and methods for sorting and/or identifying live cells. The compositions and methods provide for staining of live cells with aptamer so particular cells can be identified within or sorted from a heterogeneous population of live cells and subsequent reversal of the staining to prepare sorted and/or identified cells in their native state.

Abstract: The present disclosure relates to miRNA expression constructs, such as for expression of multiple miRNAs and use thereof to knockdown target gene expression. In some aspects, the expression constructs include a promoter element, a spacer sequence and a miRNA coding sequence. In some aspects, constructs provide enhanced immune cell function.

Abstract: The present invention provides a method for producing a nucleic acid molecule that can obtain a nucleic acid molecule that binds to a target and does not inhibit a function of the target. The production method for a nucleic acid molecule of the present invention is a method for producing a nucleic acid molecule that binds to a first biological molecule and does not inhibit a function of the first biological molecule, the method including the steps of: (A) bringing a candidate nucleic acid molecule into contact with the first biological molecule to select a nucleic acid molecule that has bound to the first biological molecule as a first selected nucleic acid molecule; and (B) selecting the first selected nucleic acid molecule as an intended nucleic acid molecule.

Abstract: Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.

Abstract: Disclosed herein is a method of inhibiting T Follicular Helper (TFH) cell-mediated differentiation and/or activation in a subject. This method involves administering to a subject in need of treatment for an autoimmune disorder a eukaryotic translation initiation factor 4E (eIF4E) inhibitor to inhibit TFH cell-mediated differentiation and/or activation in the subject. Also disclosed is a method of inhibiting T Follicular Helper (THF) cell differentiation or TFH cell activity.

Abstract: 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.

Abstract: Disclosed herein are antisense compounds and methods for decreasing Tau mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Tau-associated diseases, disorders, and conditions.

Abstract: An oligo- or polynucleotide analogue having one or more structures of the general formula: where B is a pyrimidine or purine nucleic acid base, or an analogue thereof, is used for treating obesity-related metabolic diseases.

Abstract: Oligonucleotides are provided herein that inhibit NR1H3 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with NR1H3 expression.

Abstract: Reagents and methods to cloak and uncloak RNA polymers and applications thereof are provided. Photocloaking molecules are used to label RNA polymers. Radiant energy is used to remove photoreleaseable protecting adducts and revert a RNA polymer to its native form.

Abstract: The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to ATXN3 pre-mRNA sequences, which are capable of inhibiting the expression of ATXN3 protein. Inhibition of ATXN3 expression is beneficial for the treatment of spinocerebellar ataxia.

Abstract: The present invention relates generally to methods and compositions for gene therapy and immunotherapy that activate gamma delta T-cells, and in particular, can be used in the treatment of various cancers and infectious diseases.

Abstract: Provided herein are methods for decreasing LRRK2 mRNA expression. Such methods are useful to ameliorate LRRK2 associated diseases. Such LRRK2 associated diseases include Parkinson's Disease, including non-LRRK2 mediated Parkinson's Disease.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.

Abstract: The present invention relates generally to methods and compositions for gene therapy and immunotherapy that activate gamma delta T-cells, and in particular, can be used in the treatment of various cancers and infectious diseases.

Abstract: Compositions and methods disclosed herein can help provide improved delivery of non-natural therapeutic nucleotides for the treatment of diseases such as cancer. An example composition includes an assembly of amphiphilic polynucleotides, where each amphiphilic polynucleotide includes an aptamer portion, a first nucleotide portion, and a second nucleotide portion.

Abstract: Provided herein are aptamers for binding lipoproteins, systems for binding lipoproteins, and methods of detecting lipoproteins in a sample. The aptamers are useful for detecting the levels of lipoproteins in a biological sample and selectively detecting LDL particles in the presence of HDL particles. The aptamers can also be used as therapeutic agents against various diseases.