1269 results about "Peptide Aptamers" patented technology

The present invention relates to novel cationic lipids, transfection agents, microparticles, nanoparticles, and short interfering nucleic acid (siNA) molecules. The invention also features compositions, and methods of use for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity in a subject or organism. Specifically, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver biologically active molecules, such as antibodies (e.g., monoclonal, chimeric, humanized etc.), cholesterol, hormones, antivirals, peptides, proteins, chemotherapeutics, small molecules, vitamins, co-factors, nucleosides, nucleotides, oligonucleotides, enzymatic nucleic acids, antisense nucleic acids, triplex forming oligonucleotides, 2,5-A chimeras, dsRNA, allozymes, aptamers, decoys and analogs thereof, and small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), short hairpin RNA (shRNA), and RNAi inhibitor molecules, to relevant cells and/or tissues, such as in a subject or organism. Such novel cationic lipids, microparticles, nanoparticles and transfection agents are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism. The compositions described herein are generally referred to as formulated molecular compositions (FMC) or lipid nanoparticles (LNP).

This invention features permeability enhancer molecules, and methods, to increase membrane permeability of various molecules, such as nucleic acids, polynucleotides, oligonucleotides, enzymatic nucleic acid molecules, antisense nucleic acid molecules, 2-5A antisense chimeras, triplex forming oligonucleotides, decoy RNAs, dsRNAs, siRNAs, aptamers, or antisense nucleic acids containing nucleic acid cleaving chemical groups, peptides, polypeptides, proteins, carbohydrates, steroids, metals and small molecules, thereby facilitating cellular uptake of such molecules.

Materials and methods are provided to modulate, in a controlled manner, the intracellular deliver of therapeutic agents, including therapeutic oligonucleotides using nucleic acid aptamers.

The invention provides a microsphere formulation for the sustained delivery of an aptamer, for example, an anti-Vascular Endothelial Growth Factor aptamer, to a preselected locus in a mammal, such as the eye. In addition, the invention provides methods for making such formulations, and methods of using such formulations to deliver an aptamer to a preselected locus in a mammal. In particular, the invention provides a method for delivering the aptamer to an eye for the treatment of an ocular disorder, for example, age-related macular degeneration.

The present invention provides systems, methods, and compositions for targeted delivery of a therapeutic agent organs, tissues, cells, extracellular matrix components, and intracellular compartments. The present invention provides a complex comprising a therapeutic or diagnostic agent and a nucleic acid targeting moiety, wherein the agent non-covalently associates with base pairs of the nucleic acid targeting moiety. The invention provides targeted particles comprising a particle and an inventive complex. The present invention provides methods of designing, manufacturing, and using inventive complexes and targeted particles.

The invention generally provides molecular biosensors. In particular, the invention provides molecular biosensors having one or more aptamers. The molecular biosensors are useful in several methods including in the identification and quantification of target molecules.

A method for detecting compounds of interest in bodily fluids, including exhaled breath and blood. The present invention uses biosensors that mimic naturally occurring cellular mechanisms, including RNA oligonucleotide chains or “aptamers,” in combination with signaling agents or nanotechnology to provide an effective and efficient method for diagnosing a condition and/or disease within a patient. The subject invention also provides a method for screening those analytes/biomarkers likely to be present in exhaled breath.

A method for detecting compounds of interest in bodily fluids, including exhaled breath and blood. The present invention uses biosensors that mimic naturally occurring cellular mechanisms, including RNA oligonucleotide chains or “aptamers,” in combination with molecular beacons or nanotechnology to provide an effective and efficient method for diagnosing a condition and/or disease within a patient. The subject invention also provides a method for screening those analytes/biomarkers likely to be present in exhaled breath.

Materials and methods are provided for producing and using aptamers useful as oncology therapeutics capable of binding to PDGF, PDGF isoforms, PDGF receptor, VEGF, and VEGF receptor or any combination thereof with great affinity and specificity. The compositions of the present invention are particularly useful in solid tumor therapy and can be used alone or in combination with known cytotoxic agents for the treatment of solid tumors. Also disclosed are aptamers having one or more CpG motifs embedded therein or appended thereto.

Methods of treating complement-mediated ocular disorders by administering agents that inhibit a subject's complement component in an amount sufficient to treat the ocular disorder wherein, in a selected embodiment, said agent is an anti-complement aptamer that, in a preferred embodiment, is an anti-C5 aptamer.

The present invention provides a small (business card-sized) disposable mesofluidic or microfluidic plastic cartridge containing several straight microchannels potentially filled with culture media, solubilizing reagents (e.g., detergents) nitrocellulose paper strip, gel or other matrix materials and lyophilized paramagnetic microbeads or microparticles coated with antibodies, nucleic acid aptamers, oligonucleotides, or other types of proteins or other receptors for capture, concentration and ultrasenstive detection of target analytes in environmental, food, animal, or clinical body fluid samples.

The present invention relates to compositions that can detect the presence of specific entities or substances in an environment, and provide an amplified response to the detection as manifested by release of enzymes, reporter signals or drugs. The detection and response is based on nucleic acid functionalities, such as aptamer regions that are designed to specifically bind almost any entity or ligand, and enzymatic regions that can cleave nucleic acids at specific sequences. The response can be amplified on a first order through creating an allosteric relationship between the different nucleic acid functionalities present on the same nucleic acid molecule and on a second order through the release of active cargo molecules capable of generating molecules detectable by their color, fluorescence, luminescence, or ability to modulate an electric signal.

The present invention related to fluoroalkoxy (“—OCF3”) nucleosides, nucleotides, and polynucleotides comprising fluoroalkoxy nucleotides. The present invention also relates to methods of synthesizing fluoroalkoxy nucleosides, nucleotides, and polynucleotides comprising fluoroalkoxy nucleotides. The present invention also relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity. The invention also relates to fluoroalkoxy modified nucleic acid molecules, such as ribozymes, antisense, aptamers, decoys, triplex forming oligonucleotides (TFO), immune stimulatory oligonucleotides (ISO), immune modulatory oligonucleotides (IMO), and small nucleic acid molecules, including short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against polynucloetide targets. Such small nucleic acid molecules are useful, for example, in providing compositions to treat, prevent, inhibit, or reduce diseases, traits, or conditions in a subject or organism.

Screening of a library of particles in vivo and/or in vitro using Polyplex Iterative Combinatorial Optimization (PICO) allows for the design of particles for targeting a specific organ, tissue (e.g., cancer), or cell. Particles may, for example, include different targeting agents (e.g., aptamers or plurality of aptamers) on their surfaces, and the aptamer or aptamers may be evolved to provide better targeting of the particles. Libraries of particles are enriched in characteristics of particles that have been found to migrate to a tissue of interest, be taken up by cells, etc. The process may be repeated to engineer particles of a desired specificity or biological function.

The invention discloses a Non-Equilibrium Capillary Electrophoresis of Equilibrium Mixtures (NECEEM) method and NECEEM-based practical applications. The NECEEM method is a homogeneous technique, which, in contrast to heterogeneous methods, does not require affixing molecules to a solid substrate. The method of the invention facilitates 3 practical applications. In the first application, the method allows the finding of kinetic and thermodynamic parameters of complex formation. It advantageously allows for revealing two parameters, the equilibrium dissociation constant, K_d, and the monomolecular rate constant of complex decay, k_off, in a single experiment. In the second practical application, the method of this invention provides an approach for quantitative affinity analysis of target molecules. It advantageously allows for the use of affinity probes with relatively high values of k_off. In the third practical application, the method of this invention presents a new and powerful approach to select target-binding molecules (ligands) from complex mixtures. Unique capabilities of the method in its third application include but not limited to: (a) the selection of ligands with pre-determined ranges of kinetic and thermodynamic parameters of target-ligand interactions, (b) the selection of ligands present in minute amounts in complex mixtures of biological or synthetic compounds such as combinatorial libraries of oligonucleotides, and (c) the selection of ligands for targets available in very low amounts. In particular, the method of this invention provides a novel approach for the selection of oligonucleotide aptamers. The NECEEM-based method can be used for discovery and characterization of drug candidates and the development of new diagnostic methods.

A methodology for bioassays and diagnostics in which a particulate label (ranging in size from nm-scale molecular assemblages to organisms on the scale of tens or hundreds of microns), such as, but not limited to, nanoparticles, bacteria, bacteriophage, Daphnia, and magnetic particles, serve carriers for analytes bound by molecular recognition elements such as antibodies, aptamers, etc. The described methodology is generally applicable to most pathogen assays and molecular diagnostics and also leads to enhanced sensitivity and convenience of use.

Some aspects of this disclosure provide compositions, methods, systems, and kits for controlling the activity and/or improving the specificity of RNA-programmable endonucleases, such as Cas9. For example, provided are guide RNAs (gRNAs) that are engineered to exist in an “on” or “off” state, which control the binding and hence cleavage activity of RNA-programmable endonucleases.