The present invention is a method for measuring the amount of at least one molecule in a biological sample, the method comprising a) combining the sample, or a derivative thereof, with one or more aptamers and allowing one or more molecules in the sample to bind to the aptamer(s); b) separating bound from unbound molecules; and c) quantifying the molecule(s) bound to the or each aptamer, wherein quantification of the bound molecule(s) is carried out by sequencing at least part of the or each aptamer. Uses of and products derived from the method are also contemplated.

Provided are a nucleic acid with improved ability to inhibit the expression of the MEX3B gene with low occurrence of cytotoxicity side effects, an MEX3B gene expression inhibiting agent that includes the nucleic acid, a method for inhibiting MEX3B gene expression, and a prophylactic or therapeutic drug for a disease caused by MEX3B gene expression. The present invention is a nucleic acid that is any of (1) to (3): (1) an oligonucleotide that comprises the nucleotide sequence represented by SEQ ID NO: 1 or 2; (2) an antisense oligonucleotide that inhibits expression of the MEX3B gene and that comprises a nucleotide sequence derived by deletion, substitution, and/or addition of one or two nucleotides in the nucleotide sequence represented by SEQ ID NO: 1 or 2; and (3) an antisense oligonucleotide that inhibits expression of the MEX3B gene and that includes the nucleotide sequence represented by SEQ ID NO: 3.

Provided are compositions comprising an oligonucleotide that targets Thymic stromal lymphopoietin (TSLP). The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating an airway disorder by providing an oligonucleotide that targets TSLP to a subject in need thereof. In some embodiments, the oligonucleotide targeting is specific for a long isoform of TSLP (lfTSLP).

Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for TTR.

The disclosure provides tandem arrays of CRISPR RNAs and methods of use.

The present invention relates to antisense oligonucleotides that are complimentary to SOD1, leading to decreased expression of SOD1. Reduced expression of SOD1 is beneficial in medical disorders such as Amyotrophic Lateral Sclerosis.

The present invention relates to oligomeric nucleic acid molecules and uses thereof for treating hearing loss. The present invention relates to small activating nucleic acid molecules for treating hearing loss. The small activating nucleic acid molecules of the present invention can be double-stranded or single-stranded RNA molecules targeting the promoter region of an ATOH1 gene comprising a first nucleic acid strand and a second nucleic acid strand. The double-stranded RNA molecule targeting the promoter region of the ATOH1 gene comprises two nucleic acid strands of 16 to 35 nucleotides in length, wherein one nucleic acid strand has at least 75% homology or complementarity to a target selected from the promoter region of the ATOH1 gene. The present invention also relates to pharmaceutical compositions comprising the small activating nucleic acid molecules and optionally, a pharmaceutically acceptable carrier, and methods for upregulating the expression of the ATOH1 gene and ATOH1 protein in a cell and treating diseases or conditions, related to insufficient or decreased expression of the ATOH1 gene by using the small activating nucleic acid molecules, or the pharmaceutical composition.

The embodiments disclosed herein utilized RNA targeting effectors to provide a robust CRISPR-based diagnostic with attomolar sensitivity. Embodiments disclosed herein can detect broth DNA and RNA with comparable levels of sensitivity and can differentiate targets from non-targets based on single base pair differences. Moreover, the embodiments disclosed herein can be prepared in freeze-dried format for convenient distribution and point-of-care (POC) applications. Such embodiments are useful in multiple scenarios in human health including, for example, viral detection, bacterial strain typing, sensitive genotyping, and detection of disease-associated cell free DNA.

The present embodiments provide methods, compounds, and compositions useful for inhibiting or reducing SARS-CoV-2 replication, infectivity, viral titer, or viral load, which can be useful for preventing or treating COVID-19 in an individual.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.