39 results about "Small hairpin RNA" patented technology

The present invention provides novel, stable nucleic acid-lipid particles comprising one or more Dicer-substrate dsRNAs and/or small hairpin RNAs (shRNAs), methods of making the particles, and methods of delivering and/or administering the particles (e.g., for the treatment of a disease or disorder). In some embodiments, the nucleic acid-lipid particles of the invention comprise Dicer-substrate dsRNAs and/or shRNAs. In other embodiments, the nucleic acid-lipid particles of the invention comprise Dicer-substrate dsRNAs and/or shRNAs in combination with one or more additional interfering RNAs (e.g., siRNA, aiRNA, and/or miRNA). In further embodiments, the Dicer-substrate dsRNAs and/or shRNAs present in the nucleic acid-lipid particles of the invention are chemically synthesized.

The invention provides a vector comprising an AAV-shRNA vector. The vector is preferably rAAV-151i/1694i. The invention also provides a method of suppressing or inhibiting HBV replication in liver cells infected therewith, comprising administering an amount of an AAVB-shRNA vector effective to suppress, inhibit or reduce HBV replication.

Methods, compositions, and kits that include small hairpin RNA (shRNA) useful for inhibition of gene expression, such as viral-mediated gene expression, are described.

The present invention relates to a method and composition for generating a non-naturally occurring intron and its components capable of being processed into small hairpin RNA (shRNA) and/or microRNA (miRNA) molecules by skin cells and thus inducing specific gene silencing effects on skin pigment-related genes and/or aging-causing genes in the cells. The gene silencing effects so obtained are not only useful for lightening and whitening skin colors but also useful for suppressing unwanted aging gene activities in skins.

The invention belongs to the technical field of biomedicine, and relates to a detection, treatment and prognosis target of depression and applications. An accumbens nucleus HDAC7 in neurons can be inhibited by a small hairpin RNA in an HDAC7-shrna lentivirus, and the sequence of the small hairpin RNA is 5'-GAC AAG AGC AAG CGA AGT G-3'. Experiments in the invention confirm that the HDAC7 can regulate and control the expression of many genes closely related to depression-like behavior and can be used as the detection, treatment and prognosis target of the depression; and the target provides a novel idea for the treatment of the depression, and provides a novel idea for the research and development of drugs for preventing and treating the depression.

The invention is directed to a method of characterizing a mechanism of action of an agent (e.g., a chemotherapeutic agent, a genotoxic agent). The method comprises contacting a plurality of populations of cells with an agent to be assessed, wherein each population of cells have one gene of interest targeted by a small hairpin RNA (shRNA) and wherein said gene of interest regulates cell death and a plurality of genes that regulate cell death are targeted in the plurality of populations of cells. A responsiveness of each population of cells to the agent is determined, thereby obtaining an shRNA signature of the agent, so as to identify one or more genes that mediate a response to the agent, thereby characterizing the mechanism of action of the agent. The invention is also directed an article of manufacture for characterizing a mechanism of action of a chemotherapeutic or genotoxic agent.

The invention discloses a tri-cistron muscle specific two-way co-expression gene transferant and a preparation method thereof. The gene transferant comprises an MARs cow matrix attachment region, short hairpin RNA (shRNA) taking the third exon (926-947 loca) of cow muscle colyone as a target spot, an optimally synthesized muscle specific promoter (MRF4), a humanization n-3 desaturase gene (FAD3), an internal ribosome entry site (IRES), a red fluorescent protein gene (DsRed) and a termination signal region (SV40polyA), and the independent gene transferant does not have a pronucleus main vector sequence. According to the tri-cistron muscle specific two-way co-expression gene transferant and the preparation method, an RNA interference technology is firstly combined with the muscle specific overexpression FAD3 and DsRed, therefore, gene knock-down and inverse overexpression of related genes can be performed simultaneously, and a novel thought and approach can be supplied for safely and efficiently improving characters, such as muscle and disease related characters, controlled by polygenes.

The invention discloses a bicistronic co-expression gene transfer body and a preparation method. The gene sequence of the gene transfer body is SEQ ID NO: 17. The gene transfer body comprises bovine matrix attachment regions (MARs), an RNA polymerase promoter III (U6), short hairpin RNA (shRAN) targeting the third exon of bovine myostatin (MSTN), a CMV promoter, a bovine follistatin (FSTN) and an SV40polyA signal region from the five prime end to the three prime end in order. The gene transfer body provided by the invention doesn't contain a vector backbone sequence and is a clean and safe gene transfer; and the gene transfer body combines the MAR with a knock-down vector for the first time and can effectively enhance the knock-down effect of the vector. Besides, the gene transfer body achieves the purpose of conducting knock-down and over-expression simultaneously by independently starting double promoters, and provides new ideas and paths for improving the characteristics (such as economic characteristics) of double-gene/multi-gene control.

The invention discloses a small-hairpin RNA recombinant carrier expressed by targeted inhibition integrin beta 1, which includes a basic sequence, a resistant gene sequence, a polyclonal locus sequence, a promoter sequence of an RNAi-Ready pSIREN-RetroQ-ZsGreen1 carrier, and an oligonucleotide chain of a targeted integrin beta 1 gene. The polyclonal locus includes a BamH I enzyme digestion locus and an EcoR I enzyme digestion locus. The oligonucleotide chain is composed of the BamH I enzyme digestion locus, a target sequence positive-sense strand, a stem-loop structure, a target sequence antisense strand, and the EcoR I enzyme digestion locus. The oligonucleotide chain is inserted into the polyclonal locus in the forward direction. The invention belongs to the technical field of gene engineering. The small-hairpin RNA recombinant carrier can targetedly inhibit expression of the integrin beta 1, has high specificity, high-effective and stable expression and high transfection efficiency.

The present invention relates to a method and composition for generating a non-naturally occurring intron and its components capable of being processed into small hairpin RNA (shRNA) and/or microRNA (miRNA) molecules by skin cells and thus inducing specific gene silencing effects on skin pigment-related genes and/or aging-causing genes in the cells. The gene silencing effects so obtained are not only useful for lightening and whitening skin colors but also useful for suppressing unwanted aging gene activities in skins.

RNA interference (RNAi) has become a powerful tool for blocking gene expression in mammals and mammalian cells. This approach requires the delivery of small interfering RNA (siRNA) either as RNA itself or as DNA, using an expression plasmid or virus and the coding sequence for small hairpin RNAs that are processed to siRNAs. Current expression systems for the production of siRNA in vivo rely on RNA polymerase III promoters. These are difficult to regulate and leave most of the RNA in the nucleus of the cell where it is inactive for RNA interference. The invention provides a DNA cassette for the cloning of small hairpin sequences which permit their expression and processing using RNA polymerase II. This system enables efficient transport of the pre-siRNAs to the cytoplasm where they are active and permits the use of regulated and tissue specific promoters for gene expression.

This invention relates generally to the use of ITK (IL-2 inducible T-cell kinase) inhibitors for the treatment of patients who have melanoma or other solid tumors. Applicants demonstrate that ITK lentiviral small hairpin RNA knockdowns and a small molecule inhibitor can each decrease the proliferation and migration of melanoma cell lines and a small molecule inhibitor can be used to inhibit the growth of melanomas in vivo.

The invention provides a method for constructing a targeting ROR1 CAR-T cell by using shRNA (short hairpin ribonucleic acid) to silence a human Tim-3 gene and application of the targeting ROR1 CAR-T cell. The HKP55 CAR-T cell prepared by the method can be applied to cancer treatment. According to the invention, three different targeting Tim-3 genes with small hairpin RNA (shRNA) sequence specificity are designed, and the shRNA-3 sequence with the best silencing effect is obtained through screening. Therefore, the shRNA-3 sequence and the CAR sequence of the targeted ROR1 are co-transfected into the T cell, and the ROR1 CAR-T (HKP55 CAR-T) cell with the silent Tim-3 gene is prepared. The Tim-3 expression level of HKP55 CAR-T cells is greatly reduced, and compared with ROR1 CAR-T (HKP69 CAR-T) cells, the in-vitro killing of the ROR1 CAR-T cells on target cells with high expression of Galectin-9 and cell proliferation dependent on the target cells are remarkably enhanced through silencing of the Tim-3 gene. Therefore, the HKP55 CAR-T cell can overcome a complex immunosuppressive tumor microenvironment, continuously proliferate and exert an anti-tumor effect function.

This disclosure concerns compositions and methods for novel RNA inhibition molecules (e.g., small/short hairpin RNA (shRNA) molecules), polynucleotides encoding such molecules, use of such novel RNA inhibition molecules to inhibit a target gene by suppressing the expression of the mRNA of the target gene, and for application such as the of control insect pests, and transgenic plants that produce,and are protected, by these novel RNA inhibition molecules are described.

A fusion molecule is provided that includes one or more inhibitory nucleic acids, a targeting polypeptide, and a nucleic acid binding moiety. The targeting polypeptide and the nucleic acid binding moiety include specific the amino acid sequences. A fusion molecule is also provided that includes one or more inhibitory nucleic acids, a targeting polypeptide, and a nucleic acid binding moiety adapted to bind a double-stranded RNA or to a small hairpin RNA. The targeting polypeptide being IL6 or IL21 or a fragment thereof.