Provided herein are Factor V/Factor Va-targeting aptamer compositions and antidote compositions targeting such aptamer compositions. Methods for preventing blood clots using such compositions are also provided.

The present disclosure relates to RNA aptamers and uses thereof, in particular, aptamers which specifically bind to CD133 and which demonstrate superior tumor penetration.

The present disclosure relates to compositions and methods for the regeneration and/or restoration of hair cells utilizing a composition or an agent that decreases expression of a gene in a tissue of the inner ear and a second agent.

DNA aptamers are high affinity ligands selected by genetic enrichment techniques to bind to specific protein targets. Because these represent chemically stable and reproducible molecules, they have application as affinity reagents and/or therapeutic drugs to affect the target protein's actions. NF-kB is an important mediator of the innate immune response and mediator of tissue inflammation. Although RNA and double stranded DNA aptamers have been identified to bind to the NF-kB family of proteins, the present invention represents the first identification of single stranded DNA aptamers that recognize NFkB RelA. The aptamers disclosed herein bind to several distinct regions of RelA and may be useful to antagonize the DNA binding of RelA as an inhibitor of cellular inflammation, visualize the location or amount of RelA in tissues from pathological conditions, or to quantitatively measure the activated state of RelA by affinity binding.

This invention is about the selection and development of aptamers that specifically bound HSA and GHSA. HSA and GHSA are associated with diabetes mellitus. The length of selected aptamers are around 46-106 bases, in which aptamers against HSA are consisting of 46-106 bases and aptamers against GHSA are consisting of 49-71 bases. All selected aptamers against HSA and GHSA can be potentially applied for detection and monitoring of diabetes mellitus in combination with blood glucose and HbA1C level. They also can applied in the drug development and drug delivery system in the diabetes mellitus and Alzheimer disease. In addition, chemical or fluorescence labeled these aptamers can be used for study function and location of HSA and GHSA.

An approach for modifying multiple types of bacteria to produce surface modifications that enhance the immunologic response when used as a vaccine. A series of plasmids (pYF, pYFC, pYFP, pSF, pSPF, and pSCF) may be used to transform bacteria which then produce surface-exposed ligands that bind to complement receptors on antigen presenting cells. When modified bacteria are used as a vaccine, the vaccine recipients produce significantly higher titers of specific antibodies and are better protected against challenges from the disease-causing bacteria.

This invention provides a method for the in vivo delivery of oligonucleotides. The invention utilizes the presence of one or plurality of HES linked to an oligonucleotide to deliver a nucleic acid sequence of interest into the cytoplasm of cells and tissues of live organisms. The delivery vehicle is nontoxic to cells and organisms. Since delivery is sequence-independent and crosses membranes in a receptor-independent manner, the delivered oligonucleotide can target complementary sequences in the cytoplasm as well as in the nucleus of live cells. Sequences of bacterial or viral origin can also be targeted. The method can be used for delivery of genes coding for expression of specific proteins, antisense oligonucleotides, siRNAs, shRNAs, Dicer substrates, miRNAs, anti-miRNAs or any nucleic acid sequence in a living organism. The latter include mammals, plants, and microorganisms such as bacteria, protozoa, and viruses.

The present disclosure relates to a method for preventing spontaneous oxidation of an antioxidant, material thereof and uses thereof. More particularly, the present disclosure relates to a method for preventing oxidation of an antioxidant using aptamer specifically binding to its target antioxidant and aptamer-based control of the release rate of active ingredient in the hydrogel. Aptamer having such activity can have versatile applications such as cosmeceuticals and health beverages. As an example, we provide in the present disclosure the establishing method of aptamer targeting for Vitamin C and verification of its prevention of spontaneous oxidation of Vitamin C. We also provide the detailed method for trapping such aptamer-active ingredient complex in the hydrogel. It is expected that an aptamer-trapped hydrogel of the present disclosure has functions of controlling a release rate of the active ingredient through the aptamer-based sensing of specific substance released from the skin according to the skin conditions.

The present disclosure provides compositions and methods for conducting prime editing of a target DNA molecule (e.g., a genome) that enables the incorporation of a nucleotide change and/or targeted mutagenesis. The nucleotide change can include a single-nucleotide change (e.g., any transition or any transversion), an insertion of one or more nucleotides, or a deletion of one or more nucleotides. More in particular, the disclosure provides fusion proteins comprising nucleic acid programmable DNA binding proteins (napDNAbp) and a polymerase (e.g., reverse transcriptase), which is guided to a specific DNA sequence by a modified guide RNA, named an PEgRNA. The PEgRNA has been altered (relative to a standard guide RNA) to comprise an extended portion that provides a DNA synthesis template sequence which encodes a single strand DNA flap, which is homologous to a strand of the targeted endogenous DNA sequence to be edited, but which contains the desired one or more nucleotide changes and which, following synthesis by the polymerase (e.g., reverse transcriptase), becomes incorporated into the target DNA molecule. Also disclosed herein are various methods that leverage prime editing, including treating trinucleotide repeat contraction diseases, installing targeted peptide tags, treating prion disease through the installation of protection mutations, manipulating RNA-encoding genes for the installation of RNA tags for controlling the function and expression of RNA, using prime editing to construct sophisticated gene libraries, using prime editing to insert immunoepitopes into proteins, use of prime editing to insert inducible dimerization domains into protein targets, and delivery methods, among others.

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