55 results about "RNA silencing" patented technology

The present invention is based, in part, on the discovery that endogenous mRNAs can be recruited for translational repression of target mRNAs. The RNA-silencing agents and the methods described herein, thereby provide a means by which to treat genetic (e.g., genetic neurodegenerative diseases such as Huntington's Disease) or non-genetic diseases by, for example, blocking the synthesis of proteins that contribute to the diseases. Accordingly the RNA-silencing agents of the present invention have an mRNA targeting moiety, a linking moiety, and an mRNA recruiting moiety.

The present invention relates to gene silencing, and in particular to compositions of microRNA sequences and vectors and to methods of synthesizing such in vitro and in vivo, and to methods of using such to regulate gene expression.

The present invention relates to gene silencing, and in particular to compositions of microRNA sequences and vectors and to methods of synthesizing such in vitro and in vivo, and to methods of using such to regulate gene expression.

Conserved consensus sequences from known hepatitis B virus strains and known hepatitis C virus strains, which are useful in inhibiting the expression of the viruses in mammalian cells, are provided. These sequences are useful to silence the genes of HBV and HCV, thereby providing therapeutic utility against HBV and HCV viral infection in humans.

The invention features compositions and methods for delivering small interfering (siRNAs), e.g., shRNAs, to host cells using non-pathogenic strains of Salmonella bacteria containing nucleic acid expression constructs encoding shRNAs. In this process, shRNA expressed by the Salmonella silences or knocks down genes of interest (target genes) inside target cells. The nucleic acid expression constructs of the invention include an RNA polymerase (e.g., a T7 polymerase), an RNA polymerase promoter (e.g., a T7 polymerase promoter), and an RNA polymerase terminator (e.g., a T7 polymerase terminator). The Salmonella bacteria can also include, on the same or different nucleic acid construct, an endosomal release factor (e.g., HlyA).

The invention belongs to the field of plant biotechnology, in particular provides a polygalacturonase arrestin gene CaPGIP1 cloned from hot pepper, the gene can specially inhibit the activity of polygalacturonase (PGs) secreted by pathogenic bacteria. The invention provides the function verification techniques based on the coding of the gene, such as protein making, enzyme activity testing, gene silencing, transgenic excessive expression and the like and the application of the techniques. Based on the gene and protein operation technology, the CaPGIP1 gene is proved to effectively participate in the defense reaction of hot pepper under the induction of a stress facto, and can inhibit the activity of polygalacturonase (PGs) secreted by various pathogenic bacteria. The gene silence and transgenic technology are further used for proving that the silence or less expression of the gene in the hot pepper reduces the disease resistance of a host, and the excessive expression of the gene in the transgenic tobacco can enhance the resistance; therefore, the gene codes an important disease-resistance related protein. The invention provides an important technological reserve for transforming the polygalacturonase arrestin (PGIP) gene and cultivating a new disease-resistant variety.

This invention provides UNA oligomers for gene silencing with reduced off-target effects. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.

This invention provides UNA oligomers for selectively inhibiting V30M TTR expression, which can be used in treating amyloidosis. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis by administering a UNA oligomer to a subject.

This invention provides UNA oligomers for selectively inhibiting V30M TTR expression, which can be used in treating amyloidosis. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis by administering a UNA oligomer to a subject.

The present invention discloses that vertebrate viruses encode RNA silencing suppressors / expressional enhancers which enable them to overcome host intracellular defense responses. This has paved the way for the development of protein production systems and for production systems of (recombinant) virus particles, and of vaccines directed against vertebrate viruses.

The present invention relates to the discovery of a method for inhibiting RNA silencing in a target sequence-specific manner. RNA silencing requires a set of conserved cellular factors to suppress expression of gene-encoded polypeptide. The invention provides compositions for sequence-specific inactivation of the RISC component of the RNA silencing pathway, and methods of use thereof. The RISC inactivators of the present invention enable a variety of methods for identifying and characterizing miRNAs and siRNAs, RISC-associated factors, and agents capable of modulating RNA silencing. Therapeutic methods and compositions incorporating RISC inactivators and therapeutic agents identified through use of RISC inactivators are also featured.

The present invention provides recombinant DNA constructs for inactivation of viral or endogenous genes in a cell, wherein the construct comprises viral sequence sufficient to activate RNA silencing. In another aspect, the invention provides methods for identifying RNA silencing suppressors by sequence analysis and functional tests. In yet another aspect, the invention provides a method for identifying inhibitors of RNA silencing suppressors. In still other aspects, the invention comprises methods for identifying genes in the antiviral RNA silencing pathway, enhancers of the antiviral pathway, and methods of treating or preventing viral infections using enhancers of the pathway.

The invention features compositions and methods for delivering small interfering (siRNAs), e.g., shRNAs, to host cells using non-pathogenic strains of Salmonella bacteria containing nucleic acid expression constructs encoding shRNAs. In this process, shRNA expressed by the Salmonella silences or knocks down genes of interest (target genes) inside target cells. The nucleic acid expression constructs of the invention include an RNA polymerase (e.g., a T7 polymerase), an RNA polymerase promoter (e.g., a T7 polymerase promoter), and an RNA polymerase terminator (e.g., a T7 polymerase terminator). The Salmonella bacteria can also include, on the same or different nucleic acid construct, an endosomal release factor (e.g., HlyA).

Chemically modified small interfering RNAs (siRNAs) that include both phosphorodithioate modifications (PS2-RNA) and 2′-O-methyl modifications (MePS2) provide improved RNA silencing. Specific chemically modified siRNA that show enhanced silencing of RNAs involved in resistance to chemotherapeutic agents are provided.

An expression cassette for gene silencing and replacement, including, in operable communication, a promoter, an expression attenuator, a nucleotide sequence encoding a gene for a replacement target protein, and an shRNA sequence for knockdown of an endogenous variant of the target protein, wherein the promoter, the expression attenuator, the nucleotide sequence encoding the gene for the replacement target protein, and the shRNA are expressed as a single transcript. Also included are expression vectors and cells. Also included are methods of silencing and replacement of a target gene in a cell in culture by transforming the cells with the expression vectors described herein. Also included are minimal expression cassettes suitable for therapeutic methods.

A method of enhancing expression of at least one desired gene product in a plant, comprises transgenically introducing a Tombusvirus p19 / R43W mutant gene (SEQ. ID NO: 3) into the plant, expressing at least one desired gene product in the plant, wherein expression of said product is susceptible to RNA silencing; and co-expressing the transgenically introduced p19 / R43W, to produce an amount of P19 / R43W mutant protein (SEQ. ID NO: 4) sufficient to suppress silencing of expression of said desired gene product.

Methods and compositions useful in target sequence suppression and target sequence validation are described. Polynucleotide constructs useful for gene silencing, as well as cells, plants and seeds comprising the polynucleotides and a method for using microRNAs to silence a target sequence are also described.

The present invention provides compositions and formulations that contain active compounds as RNAi inhibitors. These compositions and formulations are useful for controlling insects and pests including mosquitoes and agricultural pests.