80 results about "Small interfering RNA" patented technology

This disclosure relates to isolated oligonucleotides comprising duplex regions targeting human FXI mRNA, and delivery systems, kits and compositions comprising the same, and methods of using the same for inhibiting or downregulating FXI gene expression and treating or preventing a disease or disorder associated with aberrant or increased expression or activity of FXI or a disease or disorder where FXI plays a role.

The application provides a group of small interfering RNAs (siRNAs) targeting pyroptosis-related inflammatory reaction genes and application thereof. The pyroptosis-related inflammatory reaction genes include IL1A, IL1B, IL6, HMGB1, S100A8, S100A9 and BACH1. The application designs and synthesizes specific siRNA sequences for the above genes, and verifies that the siRNAs can efficiently and specifically inhibit the mRNA expression level of the corresponding genes through cell transfection and real-time fluorescent quantitative PCR. The application also provides a composition containing the siRNAs, a pharmaceutical composition and application thereof in the preparation of a drug for treating diseases mediated by pyroptosis-related inflammatory reaction genes (especially inflammatory diseases). The siRNAs and the composition thereof provide a new effective strategy for treating diseases related to excessive activation of pyroptosis-related inflammatory reactions, and have a wide application prospect.

The invention particularly relates to a glycosylated dendrimer for co-delivery of small interfering RNA (Ribonucleic Acid) and small molecular drugs as well as a preparation method and application of the glycosylated dendrimer. The nano platform provided by the invention is characterized in that an anti-inflammatory drug resveratrol (Res) and electrostatic adsorption small interfering RNA (si-Cp2e1) are loaded in a glycosylated dendrimer G5-Gal. The preparation method and the application comprise the following steps: preparation of G5-Gal, preparation of Res (at) G5-Gal, and preparation of Res (at) G5-Gal / si-Cp2e1. The preparation method is simple, the required raw materials are easy to obtain, the synthesis process is simple and convenient, separation and purification are easy, and the synthesized nano-drug has a liver targeting capability, can realize dual treatment of small molecule drug-small interfering RNA, and has a potential application prospect in the aspect of treatment of alcohol-related liver diseases.

Provided are compositions comprising an oligonucleotide that targets Angiopoietin-like 4 (ANGPTL4). The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating a metabolic or cardiovascular disorder by providing an oligonucleotide that targets ANGPTL4 to a subject in need thereof.

The application belongs to the technical field of gene diagnosis and treatment, and particularly relates to application of PIP4K2C gene in breast cancer detection, wherein the kit comprises a solid carrier and a detection reagent coated on the solid carrier, and the detection reagent is used for detecting PIP4K2C gene or PIP4K2C protein. The PIP4K2C gene and its protein expression product in the application can be used as a specific marker for diagnosing breast cancer. The small interfering RNA designed according to the PIP4K2C gene in the application can also be used as a gene therapy tool for treating breast cancer, thereby providing a new breast cancer treatment approach.

Disclosed herein is a composition comprising an oligonucleotide targeting MST1. The oligonucleotide may comprise a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein is a method for treating a condition associated with an MST1 mutant, comprising providing a subject with an oligonucleotide targeting MST1.

The present application relates to the application of long-chain non-coding RNA HILAR inhibitor in the preparation of drugs for preventing or / and treating lung adenocarcinoma metastasis. The present application provides specific intervention means for HILAR, and verifies the effectiveness of inhibiting lung adenocarcinoma metastasis: after the expression of HILAR is targeted and silenced in vitro by small interfering RNA technology, the invasion ability of lung adenocarcinoma cells is significantly inhibited, and the metastasis phenotype is obviously reversed; more importantly, through the delivery of short hairpin RNA by adeno-associated virus vector, the formation and development of lung metastasis are effectively reduced in a lung adenocarcinoma metastasis mouse model, and the in vivo imaging shows that the tumor luminescence signal intensity of the experimental group is continuously lower than that of the control group, and the overall survival condition of the animal is improved.

Provided are interfering RNAs such as small interfering RNAs (siRNAs) targeting the messengerRNA of adenine nucleotide translocator 2 (ANT2), pharmaceutical compositions comprising such, and therapeutic uses thereof for treating diseases associated with ANT2, for example,cancer and metabolic diseases.

The invention discloses siRNA (small interfering ribonucleic acid) targeting SOX7, application of the siRNA in preparation of a medicine for treating chronic pain and the medicine, the small interfering RNA targeting the SOX7 gene comprises the SOX7 siRNA, the positive-sense strand sequence of the SOX7 siRNA is shown as SEQ.ID.NO.1, the antisense strand sequence of the SOX7 siRNA is shown as SEQ.ID.NO.2, and the small interfering RNA can target SOX7 genes of human and mice at the same time. The SOX7 siRNA is a group of siRNA for inhibiting the expression of the SOX7 gene, and siRNA targets are designed in a conserved region of the SOX7 gene and are located in a coding region. The expression of the SOX7 gene can be effectively reduced in vivo; by inhibiting the expression of the SOX7 gene, mechanical hyperalgesia generated by SNL induction can be effectively relieved, a foundation is laid for clinical treatment of sexual pain, and the application and popularization value is great.

The application discloses application of AGPG in treatment of endocrine-resistant estrogen receptor positive breast cancer, and finds that AGPG can promote in-vitro endocrine-resistant cell cycle progression and cell proliferation through stable knockout research. Finally, a small interfering RNA drug is tested in an in-vivo experiment, and it is further proved that down-regulation of AGPG mediated by the small interfering RNA can significantly inhibit growth of tamoxifen-resistant MCF7 xenografts.

The application provides a long-chain non-coding RNA gene and a small interfering RNA thereof in the preparation of a drug for inhibiting triple-negative breast cancer. The nucleotide sequence of the long-chain non-coding RNA gene is shown as SEQ ID NO. 1. The nucleotide sequence of the small interfering RNA is shown as SEQ ID NO. 2. The application also provides a reagent for detecting the long-chain non-coding RNA gene in the preparation of a kit for diagnosing triple-negative breast cancer. The application also provides the long-chain non-coding RNA gene as a therapeutic target in the screening of a drug for treating triple-negative breast cancer. The application proves that overexpression of the long-chain non-coding RNA gene promotes the proliferation, migration and invasion of triple-negative breast cancer cells through in-vitro and in-vivo experiments; and proves that knockdown of the long-chain non-coding RNA gene can inhibit the proliferation, migration and invasion of triple-negative breast cancer cells through in-vitro and in-vivo experiments.

This disclosure relates to isolated oligonucleotides comprising duplex regions targeting human FXI mRNA, and delivery systems, kits and compositions comprising the same, and methods of using the same for inhibiting or downregulating FXI gene expression and treating or preventing a disease or disorder associated with aberrant or increased expression or activity of FXI or a disease or disorder where FXI plays a role.

This invention belongs to the field of biomedicine, specifically relating to small interfering RNA (sRNA) targeting the TNF-α gene; it includes a sense strand and an antisense strand; the sense strand and / or the antisense strand has a length ranging from 19 to 25 nucleotides, and the antisense strand is inversely complementary to a segment on the target gene. Furthermore, it achieves drug delivery of the small interfering RNA through a specific target gene, enabling precise drug targeting at the site of colonic inflammation, enhancing the therapeutic effect of IBD without causing systemic immunosuppression, thus providing a more precise and efficient gene regulation tool.

Disclosed herein are metabolizable binary gold supraclusters, which, in some implementations, comprise cationic gold nanoclusters laden with a therapeutic nucleic acid, such as a small interfering RNA (siRNA), and are intertwined through bioresponsive crosslinkers within a hydrophilic polymer matrix. Also provided herein are uses of the supraclusters, e.g., in treatment of diseases such as cancers.

Described herein are small interfering RNA (siRNA) molecules and their use in methods and pharmaceutical compositions for inhibiting the expression of EF-hand domain-containing protein 1. Also, described herein are the use of said siRNA molecules in the treatment of metabolic liver disease, metabolic dysfunction-associated steatotic liver disease, or steatohepatitis, and reduces Ca2+-induced mitochondrial fission.

Small interfering RNAs inhibiting the programmed cell death ligand 1 (PD-L1) and the use thereof. Sense strands of the small interfering RNAs (siRNAs) have a length of 18-22 bases, and the siRNAs specifically pair with a specific region of a target gene. The siRNAs have the following uses: (1) treating cancer which involves high PD-L1 expression; or (2) inhibiting the expression of PD-L1 protein.

Described herein, inter alia, are compounds for treating cancer and methods of use. This disclosure features chemical entities (e.g., small hairpin RNAs (shRNAs), micro RNA (miRNAs), small interfering RNA (siRNAs), small molecule inhibitors, antisense nucleic acids, peptides, viruses, CRISPR-sgRNAs, or combinations thereof) that inhibit one or more of m6A writers (e.g., methyltransferase like 3 (Mettl3 or MT-A70) or methyltransferase like-14 (Mettl14)), m6Am writers (e.g., phosphorylated CTD interacting factor I (PCIF 1), or Mettl3 / 14), m6A erasers (e.g., fat-mass and obesity-associated protein (FTO) or ALKB homolog 5 (ALKBH5)), m6Am erasers (e.g., FTO), m6A readers (e.g., YTH domain-containing family proteins (YTHs)), YTF domain family member 1 (YTHDF 1), YTF domain family member 2 (YTHDF 2), YTF domain family member 3 (YTHDF 3), or tyrosine-protein phosphatase non-receptor type 2 (PTPN2).

This disclosure relates to isolated oligonucleotides comprising duplex regions targeting human APP mRNA, and delivery systems, kits and compositions comprising the same, and methods of using the same for inhibiting or downregulating APP gene expression and treating or preventing a disease or disorder associated with aberrant or increased expression or activity of APP or a disease or disorder where APP plays a role.

Embodiments of the present disclosure include compositions and methods for performing in situ hybridization reactions. In particular, the present disclosure provides RNA blocking molecules that enhance detection of a target RNA molecule (e.g., an mRNA molecule, a microRNA (miRNA) molecule, a small non-coding RNA (sncRNA) molecule, a PIWI-interacting RNA (piRNA) molecule, a small interfering RNA (siRNA) molecule, and / or an anti-sense oligo (ASO) molecule) by reducing binding of a target probe to a non-target RNA molecule in a sample.

The invention relates to medical application of IRF9 in prevention and treatment of trastuzumab (TRZ) cardiotoxicity, and belongs to the technical field of biological medicine. The invention reveals that the expression of the interferon regulatory factor 9 (IRF9) in the TRZ cardiotoxic myocardial tissue is up-regulated for the first time, and myocardial damage is aggravated by promoting pyroptosis of myocardial cells. On the basis, the invention provides the IRF9 as a diagnostic marker of TRZ cardiotoxicity, and provides an application of an IRF9 low expression vector or small interfering RNA (siRNA) in preparation of drugs for preventing or treating TRZ cardiotoxicity. Animal experiments show that cardiac function decline, cardiac hypertrophy and fibrosis caused by TRZ can be remarkably improved by myocardial cell specific low-expression IRF9. The invention provides a new target spot and an intervention strategy for clinical prevention and treatment of TRZ cardiotoxicity.

A method of detecting target nucleic acids, including small non-coding RNAs (sncRNAs), microRNAs (miRNAs), small interfering RNAs (siRNAs), PIWI-interacting RNA (piRNA), or antisense oligonucleotides (ASO) molecules, in a cell, comprising a post-fixation step using an aldehyde-containing fixative after the initial fixation step prior to in situ hybridization.

This invention generally relates to a novel composition of RNA / mRNA medicines as well as vaccines produced by using replicase- and / or RNA-dependent RNA polymerase (RdRp)-mediated RNA cycling reaction (RCR). The present invention is useful for developing a variety of self-amplifying RNA / mRNA (samRNA) medicines and vaccines containing at least a replicase / RdRp-binding site in the 5′- or 3′-end, or both, of any desired RNA molecule, including but not limited to antisense RNA (aRNA), small interfering RNA (siRNA), short hairpin RNA (shRNA), microRNA (miRNA) / miRNA precursor, long non-coding RNA (lnRNA) and mRNA. These RNA molecules can be either in single-stranded or in double-stranded, or mixed, conformation. The samRNA so obtained is useful not only for producing RNA-based vaccines and / or medicines but also for generating the mRNA-associated proteins, peptides, and / or antibodies under a proper in-vitro or in-cell translation condition. The replicase / RdRp-binding sites used in samRNA are derived or modified from coronaviral (e.g. COVID-19) and / or hepatitis C viral (HCV) RNA-dependent RNA polymerases (RdRp) in either single-stranded or double-stranded compositions.

The application discloses a preparation method and application of a chemotherapy drug and small interfering RNA tumor-targeting co-delivery nano-therapeutic system, and relates to construction and preparation of a tumor-targeting nano-therapeutic system co-loading a chemotherapy drug, camptothecin, and small interfering RNA (siRac1) targeting Rac1. The application uses N,N-dimethylethylenediamine modified mesoporous polydopamine, loads camptothecin and siRac1, and is then modified by hyaluronic acid to obtain a co-delivery nano-therapeutic system with tumor active targeting. Compared with traditional chemotherapy drugs, camptothecin, the application can realize tumor active targeting drug delivery, and can inhibit tumor chemotherapy resistance mediated by Rac1 by silencing the expression of Rac1 through siRac1, so that the sensitivity of drug-resistant tumor cells to the chemotherapy drug, camptothecin, is restored, tumor drug resistance is reversed, the treatment effect of drug-resistant triple-negative drug-resistant breast cancer is significantly improved, and the application has a good clinical application prospect.

Described herein is the preparation and use of amino acid-modified lipids for delivery of a small interfering RNA (siRNA), mRNA, miRNA, shRNA, or oligonucleotide via formation of lipid nanoparticles (LNPs) comprising the siRNA, mRNA, miRNA, shRNA, or oligonucleotide and the amino acid-modified lipid. Use of the described lipid nanoparticles to silence overexpression of oncogenes is described.

The application provides a medicine for treating an immune checkpoint inhibitor-resistant tumor, comprising a polyunsaturated fatty acid, an ACLY inhibitor and an immune checkpoint inhibitor. The polyunsaturated fatty acid comprises one or more of omega-3 polyunsaturated fatty acids and omega-6 polyunsaturated fatty acids. The ACLY inhibitor is selected from one or more of the following: Bempedoic acid, SB 204990, NDI-091143, BMS-303141, small interfering RNA targeting the ACLY gene and short hairpin RNA targeting the ACLY gene. The medicine has good treatment effect on the immune checkpoint inhibitor-resistant tumor.

The invention provides a liver targeted presentation inhibition sequence combination, which comprises a small interfering RNA (Ribonucleic Acid) and a short hairpin RNA sequence as shown in SEQ ID NO.2, and the small interfering RNA comprises a small interfering RNA basic sequence as shown in SEQ ID NO.1. The invention also provides an application. The inhibition sequence combination is used for preparing a medicine for inhibiting FMO3 level and inhibiting TMA-FMO3-TMAO axis mediated bile acid metabolism and cholesterol transporter. The liver targeted presentation inhibition sequence combination has good specificity and high transfection efficiency, inhibits TMA-FMO3-TMAO axis mediated bile acid metabolism and cholesterol transporter by inhibiting the FMO3 level, has good potential for improving cholelithiasis and other related diseases, and is used for preparing cholelithiasis related drugs.

The invention relates to the technical field of biological medicines, and particularly discloses a cancer suppression small interfering RNA (Ribonucleic Acid) capable of simultaneously targeting cancer genes PTTG1 and STMN1 and application of the cancer suppression small interfering RNA, and the technical key points are as follows: the positive-sense strand sequence of the small interfering RNA is GGGAGAUCUCAAGUUUCAATT, and the antisense strand sequence of the small interfering RNA is UUGAAACUUGAGAUCUCCCTT. The small interfering RNA provided by the invention can specifically silence the expression of PTTG1 and STMN1 at the same time. Compared with siRNA (such as siRNA for ASCC3 or TRAPPC4) targeting a single gene in the prior art, the siRNA provided by the invention has the advantages that synergistic inhibition on tumor proliferation and metastasis pathways is realized through double-targeting design, and the inhibition effect on tumor cell growth is more remarkable; compared with a method of physically mixing two single siRNAs, the method provided by the invention has higher cell apoptosis induction efficiency and better in-vivo tumor inhibition effect; the small interfering RNA is effective in various liver cancer cells, has a lasting effect in an animal model, and provides a core molecular entity for developing novel antitumor drugs.

The invention provides a nucleic acid aptamer and a screening method thereof, the aptamer is of an oligonucleotide DNA structure, the nucleotide sequence of the nucleic acid aptamer is the nucleotide sequence of any DNA fragment as shown in SEQ ID NO: 1-3, and the nucleic acid aptamer can specifically target cardiac muscle cells (CMs). According to the invention, a new strategy can be provided for clinical treatment of cardiovascular diseases, and meanwhile, bioactive molecules such as microRNA (miRNA), small interfering RNA (small interfering RNA, siRNA), lipidosome, microspheres, microcapsules and the like can be carried to be used as an intracellular drug delivery tool.

A novel micropeptide, MP36, and its applications are provided. This MP36 micropeptide can be used to prepare reagents or drugs for the detection, prevention, or treatment of tumors, including one or more of lung cancer, breast cancer, colon cancer, glioma, bladder cancer, gastric cancer, head and neck cancer, and sarcoma. In tumors, the MP36 micropeptide can inhibit tumor cell growth and / or metastasis by specifically inhibiting MP36 expression via small interfering RNA; or by overexpressing MP36 to inhibit tumor cell growth and / or metastasis; or by conjugating a membrane-penetrating peptide to inhibit tumor growth and / or metastasis. It has significant value for new drug development and tumor detection.

This invention discloses a dual-pathway nanoformulation targeting the sclera, its preparation method, and its applications. The nanoformulation uses ketithial-modified fluorinated polyethyleneimine (TK-F-PEI) as a carrier, with a core loaded with hypoxia-inducible factor-1α small interfering RNA (HIF-1α siRNA) and a surface-coupled chemokine CCL2 nucleic acid aptamer. After local ocular administration, this formulation can efficiently target scleral lesions in myopic eyes, neutralizing CCL2 through the aptamer to improve the scleral inflammatory microenvironment, while simultaneously releasing siRNA to silence HIF-1α to inhibit the intracellular hypoxia pathway, synergistically reversing pathological scleral remodeling and delaying axial elongation. This invention's formulation exhibits strong targeting, good safety, and significant efficacy, providing a novel combined targeted drug delivery strategy for the clinical treatment of myopia, especially high myopia.