34 results about "Genetic modulation" patented technology

The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More particularly, the present compositions comprise one strand having an alternating motif and another strand having a hemimer motif, a blockmer motif, a fully modified motif or a positionally modified motif. At least one of the strands has complementarity to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.

Disclosed herein are Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) / CRISPR-associated (Cas) 9-based system related compositions and methods of using said CRISPR / Cas9-based system related compositions for altering gene expression and genome engineering. Also disclosed herein are compositions and methods of using said compositions for altering gene expression and genome engineering in muscle, such as skeletal muscle and cardiac muscle.

The present invention relates to the field of biotechnology or genetic engineering. More specifically, the present invention relates to a multiple inducible gene regulation system that functions within cells to simultaneously control the quantitative expression of multiple genes.

The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More particularly, the present compositions comprise one strand having a gapped motif and another strand having a gapped motif, a hemimer motif, a blockmer motif, a fully modified motif, a positionally modified motif or an alternating motif. At least one of the strands has complementarity to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In some embodiments, the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.

The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More particularly, the present compositions comprise one strand having a gapped motif and another strand having a gapped motif, a hemimer motif, a blockmer motif, a fully modified motif, a positionally modified motif or an alternating motif. At least one of the strands has complementarity to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In some embodiments, the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.

The present invention provides double stranded compositions wherein the first strand is modified to have a particular motif and the second strand is modified a selected motif. More particularly, the present compositions comprise an antisense strand that is modified to have a positional / full motif and the sense strand is modified to have an alternating motif, a hemimer motif, a blockmer motif, a gapped motif, a positional motif, a positional / full motif or a fully modified motif. Each strand further comprises one or more phosphorothioate internucleoside linkage. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes.

The present invention encompasses compositions and methods to allow one to deliver and express multiple genes under the control of multiple gene regulatory components in a recipient cell via a synthetic chromosome. The engineering of synthetic chromosomes to contain multiple gene control units permits the construction of complex biological circuits.

Provided herein are methods for inducing CRISPR / Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.

The present invention provides a method for detecting the source of miRNA, comprising the following steps: 1) providing the information of the miRNA precursor sequence to be detected in the species; 2) providing the pseudogene information of the species in the pseudogene database; 3) converting the Whether the physical position of the miRNA precursor sequence to be detected overlaps with the nucleotide sequence of the pseudogene of the species, and the pseudogene with a degree of overlap with the miRNA precursor sequence to be detected is used to determine the candidate pseudogene; 4) according to the miRNA precursor sequence to be detected Calculate the coincidence degree I of the two types of sequences at the physical position of the chromosome sequence and the pseudogene sequence of the species genome; 5) when the coincidence degree I meets the condition of 1≧1>0.3, determine that the miRNA to be detected is composed of The candidate pseudogenes were generated. The invention not only fills in the research blank of the relationship between miRNA and pseudogenes, but also can batch discover pseudogenes with potential biological functions in the genome, and provide candidate pseudogenes for the construction of gene regulation network.

The present invention provides compositions comprising at least one oligomeric compound comprising an alternating motif and further include a region that is complementary to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.

The invention described herein provides compositions and reagents for assembling a tripartite complex at a specific location of a target DNA. The invention also provides methods for using the complex to, for example, label a specific genomic locus, to regulate the expression of a target gene, or to create a gene regulatory network.

The present invention refers to gene expression cassettes encoding enzymes involved in the metabolic pathway for the biosynthesis of hemicelluloses, cellulose and / or uronic acids, and to a method for genetic transformation of plant cells through the introduction of one or more gene expression cassettes from the present invention, and the overexpression and repression of these genes in plants. More particularly, the present invention refers to a method for introducing expression cassettes in plants. Additionally, the present invention refers to genetically modified plants. The present invention also refers to the attainment of the genetically modified plant, its derived plants and seeds, as well as the wood, paper and cellulose from this plant.

The present invention is directed to novel self-forming polynucleotide nanoparticles, and the use of such nanoparticles and compositions comprising the same for gene modulation in a variety of organisms.

The invention discloses a theophylline-induced RNA disturbance on-off system and gene control method and use thereof. The theophylline-induced RNA disturbance on-off system at least comprises an RNA disturbance shutter. The RNA disturbance shutter is in itself an artificial miRNA fore-body or is transferred to an artificial miRNA fore-body. The artificial miRNA fore-body includes miRNA fore-body and the theophylline RNA aptamer of the annular section that is mixed with the miRNA fore-body; the theophylline RNA aptamer can be combined with theophylline to obstacle the interference effect of RNA. The RNA disturbance on-off system of the invention can activate or suppress RNA noise channel according to the theophylline concentration and dependence, thereby controlling the expression of target genes.

Disclosed herein are clustered regularly interspaced short palindromic repeat (CRISPR) / CRISPR-associated (Cas)9-based system-related compositions and use of said CRISPR / Cas9-based system-related compositions for altering gene expression and genome engineering The method of transformation. Also disclosed herein are compositions and methods for altering gene expression and genome engineering in muscles, such as skeletal and cardiac muscle, using the compositions.