105results about How to "Suppress gene expression" patented technology

The present invention relates to methods of treating respiratory disorders of all types, including pulmonary disorders, by delivering inhibitory nucleic acid molecules directly to the respiratory system. siRNAs or other nucleic acids are delivered to the lung / respiratory system for the treatment of disease.

Provided is a novel nucleic acid molecule that can be produced easily and efficiently and can inhibit the expression of a gene. The nucleic acid molecule is a single-stranded nucleic acid molecule including an expression inhibitory sequence that inhibits expression of a target gene. The single-stranded nucleic acid molecule includes: a region (X); a linker region (Lx); and a region (Xc). The linker region (Lx) is linked between the regions (Xc) and (Xc). The region (Xc) is complementary to the region (X). At least one of the regions (X) and (Xc) includes the expression inhibitory sequence. The linker region (Lx) has a non-nucleotide structure including at least one of a pyrrolidine skeleton and a piperidine skeleton. According to this single-stranded nucleic acid molecule, it is possible to inhibit the expression of the target gene.

The present invention provides compositions comprising one or more RNAi agents (e.g., siRNAs, shRNAs, or RNAi vectors) for the treatment of conditions and diseases mediated by (e.g., featuring IgE-mediated hypersensitivity), as well as systems for identifying RNAi agents effective for this purpose. The compositions are suitable for the treatment of allergic rhinitis and / or asthma. In certain embodiments of the invention the RNAi agent is targeted to a transcript that encodes a protein selected from the group consisisting of the FCεRIα chain, the FCεRIβ chain, c-Kit, Lyn, Syk, ICOS, OX40L, CD40, CD80, CD86, Re1A, Re1B, 4-1BB ligand, TLR1, TLR2, TLR3, TLR4, TLR5, TLR6, TLR7, TLR8, TLR9, CD83, SLAM, common γ chain, and COX-2. In addition, the invention provides RNAi agent / delivery agent compositions and methods of use. In certain embodiments of the invention compositions comprising an RNAi agent are delivered by the respiratory route.

A variety of zinc finger proteins (ZFPs) and methods utilizing such proteins are provided for use in treating chronic pain. ZFPs that bind to a target site in genes that are aberrantly expressed in subjects having chronic pain are described. In addition, ZFPs that bind to a target site in genes expressed at normal levels in subjects experiencing chronic pain, modulation of whose expression results in decreased pain perception, are also provided. For example, genes that are over-expressed in the dorsal root ganglia (DRG) of pain patients (e.g., Nav1.8) can be repressed.

The present invention relates to the identification, the isolation and the use of molecules interfering with the function(s) mediated by the poliovirus receptor (PVR) on cells. The molecules can be used for the treatment of cells having a metastatic potential, metastasis and cancer. Further methods are provided that are useful for identifying and isolating molecules which have the capacity to modulate PVR mediated adhesion or invasion potential of cells.

Novel compounds having activity as non-covalent inhibitors of urokinase and having activity in reducing or inhibiting blood vessel formation are provided. These compounds have Pi a group having an amidino or guanidino moiety or derivative thereof. These compounds are useful in vitro for monitoring plasminogen activator levels and in vivo in treatment of conditions which are ameliorated by inhibition of or decreased activity of urokinase and in treating pathologic conditions wherein blood vessel formation is related to a pathologic condition.

Endogenous gene regulation mechanisms together with microRNAs expressed in many organisms can be used to provide regulated or conditional expression of transgenes by placing an appropriate sequence, a microRNA binding site, within the transcribed gene. This microRNA-dependent transcription regulation can be further regulated using microRNA inhibitors.

The present invention provides a novel method for preventing and treating memory and cognitive impairment resulting from oxidative stress, inflammation and the process of aging, as well as, neurodegenerative conditions. The method is comprised of administering a composition comprising a mixture of Free-B-Ring flavonoids and flavans synthesized and / or isolated from a single plant or multiple plants to a host in need thereof. The present also includes a novel method for simultaneously inhibiting expression of pro-inflammatory cytokines, preventing ROS generation and augmenting anti-oxidant defenses. The activity of this composition is conducive to ultimately preserving cognitive function and providing a level of neuroprotection.

Disclosed are a protein encoded by a gene having a nucleotide sequence represented by any of SEQ ID NOs: 1 to 65 or a fragment thereof, an antibody recognizing the protein or antigen-binding fragment thereof, and a polynucleotide having a sequence comprising at least 12 consecutive nucleotides of a nucleotide sequence represented by any of SEQ ID NOs: 1 to 65 or a nucleotide sequence complementary thereto. The gene and the protein of the invention is useful for diagnosing and treating cancer.

Novel compounds having activity inhibitors of urokinase and in reducing or inhibiting blood vessel formation are provided. These compounds have an arginine or arginine mimic aldehyde or an arginine ketoamide group at P1. These compounds are useful in vitro for monitoring plasminogen activator levels and in vivo in treatment of conditions which are ameliorated by inhibition of or decreased activity of urokinase and in treating pathologic conditions wherein blood vessel formation is related to a pathologic condition.

The present invention provides improved methods of attenuating gene expression through the phenomenon of RNA interference. The invention provides methods of synthesis of double stranded RNAs (dsRNAs) of increased potency for use as small interfering RNA (siRNA). Surprisingly and unexpectedly, siRNAs made by the methods of the invention are significantly more potent than previously available siRNAs.

A novel and effective strategy for organ transplantation is provided. A therapeutic agent and method for preventing an acute rejection in a transplanted organ and improving prognosis are provided. A therapeutic agent for suppressing a rejection in organ transplantation, which comprises an NF-κB decoy compound, is provided. Representatively, the organ transplantation is kidney transplantation. The therapeutic agent further comprises an ultrasonic inspection contrast agent. A therapeutic agent for improving prognosis in organ transplantation is also provided. The therapeutic agent is administered into a donor organ. The transfection efficiency of the NF-κB decoy compound into the organ may be enhanced by ultrasound treatment.

The present invention concerns methods and reagents useful in modulating adenosine A1 receptor (ADORA1) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small interfering RNA (siRNA) molecules capable of mediating RNA interference (RNAi) against ADORA1 and related receptors.

Methods are provided for treating HIV and cancer in a subject in need thereof by administering to the subject therapeutically effective amounts of an mTOR inhibitor. Other methods are provided for treating subjects infected with HIV by administering to the subject therapeutically effective amounts of the mTOR inhibitor INK128, GSK2126458, AZD2014 or Torin-2.

The present invention relates to the identification, the isolation and the use of molecules interfering with the function (s) mediated by the poliovirus receptor (PVR) on cells. The molecules can be used for the treatment of cells having a metastatic potential, metastasis and cancer. Further methods are provided that are useful for identifying and isolating molecules, which have the capacity to modulate PVR mediated adhesion or invasion potential of cells.

Calmodulin-like polypeptides named rgs-CaM are disclosed. cDNAs coding rgs-CaM are also provided. In addition, methods of using rgs-CaM cDNAs and polypeptides for modulating gene expression in plants are also provided.

Disclosed are bottlebrush poly(ethylene glycol) (PEG) polymer-RNA conjugates (pacRNA) having a plurality of PEG side chains attached to a polymer backbone, and one or more RNA oligonucleotides attached to the polymer backbone via a cleavable linkage. The pacRNAs offer potential useful novel RNA-based therapeutics.

Provided is a novel nucleic acid molecule that can be produced easily and efficiently and can inhibit the expression of a gene. The nucleic acid molecule is a single-stranded nucleic acid molecule including an expression inhibitory sequence that inhibits expression of a target gene. The single-stranded nucleic acid molecule includes: a region (X); a linker region (Lx); and a region (Xc). The linker region (Lx) is linked between the regions (Xc) and (Xc). The region (Xc) is complementary to the region (X). At least one of the regions (X) and (Xc) includes the expression inhibitory sequence. The linker region (Lx) has a non-nucleotide structure including at least one of a pyrrolidine skeleton and a piperidine skeleton. According to this single-stranded nucleic acid molecule, it is possible to inhibit the expression of the target gene.

The invention discloses a method for preparing nanometer magnetic microspheres and an anticancer oral preparation prepared by utilizing the method. The method comprises the process steps of: a, material preparation, in which a carrier material, a framework material, a drug and a solvent are prepared; b, the preparation; c, the stability and drying; and d, the crushing and preparation and so on. The method takes beta-cyclodextrin as the framework material and includes the carrier material (a nanometer magnetic biomaterial) and drug combination and so on. The drug-loaded nanometer magnetic microspheres can quickly reach appointed focus target positions, penetrate target cell membranes to enter cells, realize the trans-membrane transport of target macromolecules, achieve the effects of targeted release and slow / controlled release, improve the drug concentration in target cells, and inhibit the gene expression and amplification of the target cells to ensure that the target cells are degenerated to necrotize or apoptosize; at the same time, the microspheres strengthen the killing activity of T cells and NK cells and the phagotrophic ability of macrophages to the target cells, improve the immunity of organisms, and achieve the effects of multiple treatment and multi-drug resistance reverse.

The present invention relates to a method of preventing or treating a disease caused by bacterial infection by administering an effective amount of a modulator of bacterial adenylyl cyclase. The invention also provides pharmaceutical compositions useful for preventing or treating a disease, with the compositions containing a therapeutically effective amount of a modulator of bacterial adenylyl cyclase. The invention also provides screening methods for identifying selective modulators of bacterial adenylyl cyclase that do not substantially modulate adenylyl cyclase of the subject. The invention also provides methods for culturing bacterial pathogens and methods for inducing the pathogenic state in vitro.

Genomic DNA and cDNA encoding GA 3β-hydroxylase were isolated from rice. When the expression of these genes was suppressed in rice plants, the plants became dwarfed compared with the wild type plants.

The present invention relates to a method of preventing or treating a disease caused by infection by a eukaryotic pathogen, wherein the method comprises administering an effective amount of a modulator of a eukaryotic pathogen's adenylyl cyclase. The invention also provides pharmaceutical compositions useful for preventing or treating a disease, with the compositions containing a therapeutically effective amount of a modulator of a eukaryotic pathogen's adenylyl cyclase. The invention also provides screening methods for identifying selective modulators of a eukaryotic pathogen's adenylyl cyclase that do not substantially modulate an adenylyl cyclase of the subject. The invention also provides methods for culturing eukaryotic pathogens and methods for inducing the pathogenic state in vitro.

The invention relates to a shRNA (Short Hairpin Ribonucleic Acid) kit for treating an animal model suffered from an alzheimer disease. The kit internally comprises shRAN slow virus expression plasmid vector concentrated liquor capable of specifically suppressing a caspase-3 gene, and shRAN slow virus expression plasmid vector concentrated liquor as negative control, wherein the shRAN comprises sequences of SEQ ID No.1 and SEQ ID NO.2 in a sequence table. Compared with other means, the shRAN sequence or a reagent obtained therefrom can specifically and conveniently suppress the expression of disease-related proteins at high efficiency when entering the body of the animal model in a certain manner and lead disease-related genes to be silent, thereby achieving the treatment purpose; and therefore, the kit provided by the invention can be used for gene therapy for the animal model suffered from the alzheimer diseases.

Provided is a novel nucleic acid molecule that is a single-stranded nucleic acid molecule including an expression inhibitory sequence that inhibits expression of a target gene. The single-stranded nucleic acid molecule includes, in sequence from the 5′ side to the 3′ side: a 5′ side region (Xc); an inner region (Z); and a 3′ side region (Yc). The inner region (Z) is composed of an inner 5′ side region (X) and an inner 3′ side region (Y) that are linked to each other. The 5′ side region (Xc) is complementary to the inner 5′ side region (X). The 3′ side region (Yc) is complementary to the inner 3′ side region (Y). At least one of the inner region (Z), the 5′ side region (Xc), and the 3′ side region (Yc) includes the expression inhibitory sequence.

The present disclosure provides a siRNA for inhibiting the expression of apolipoprotein C3 (ApoC3) gene, and a pharmaceutical composition and a conjugate comprising the siRNA; wherein each nucleotide in the siRNA is independently a modified nucleotide, and the siRNA comprises a sense strand and an antisense strand; the sense strand comprises a nucleotide sequence A, the nucleotide sequence A having the same length as the nucleotide sequence as represented by SEQ ID NO:1 with no more than 3 nucleotide differences; the antisense strand comprises a nucleotide sequence B, the nucleotide sequence B having the same length as the nucleotide sequence as represented by SEQ ID NO:2 with no more than 3 nucleotide differences.

A human anti-HBsAg mono-chain antibody / human antibody light chain constant region / protamine truncate recombinant gene, encoding protein and use are disclosed. The protein has antigen and nucleic acid combined activities. The process is carried out by: combining HBV siRNA with siRNA expression plasmid, transferring to HBV infectious cell specifically, and inhibiting HBV gene expression and copy. It has excellent specificity, efficiency, continuity and safety.

The invention provides a conjugate of functional oligonucleotide with poly(ethylene oxide), polyion complex of the conjugate with cationic polymer, and micelle thereof. The micelle improves stability of the oligonucleotide in animal cells and its specific bindability to target gene.