161 results about "Regulator gene" patented technology

The present disclosure relates to isolated nucleic acid molecules comprising at least two double-stranded inhibitory ribonucleic acid (RNA) molecules adapted to silence the same or different genes by RNA interference to enhance silence, thereby modulating gene expression.

Disclosed herein is a novel Cas9 protein. Further described herein are fusion proteins, compositions, and methods comprising the same. The novel Cas9 protein may be used, for example, in compositions and methods for modulating expression of a gene, for correcting a mutant gene, and for treating a disease.

The present disclosure generally relates to, inter alia, a class of chimeric switch receptors containing an endodomain of an IL-9 receptor, engineered to modulate transcriptional regulation in a ligand-dependent manner. The disclosure also provides compositions and methods useful for producing such receptors, nucleic acids encoding same, host cells genetically modified with the nucleic acids, as well as methods for modulating gene expression, modulating an activity of a cell, and / or for the treatment of various health conditions or diseases.

The present invention relates to an induced pluripotent stem cell (iPSC) or a somatic cell derived from an iPSC by forward programming or directed differentiation constitutively expressing an alternative splice form of Lamin A. The cells comprise an expression cassette comprising a coding sequence for the alternative Lamin A splice form operably linked to a cumate operator (CuO) sequence and a constitutive promoter. Also provided is the use of the cells described herein in a method of identifying a gene or combination of genes involved in the reversal of an ageing phenotype or in the maintenance of a non-aged phenotype. Methods of rejuvenating somatic cells, treating diseases or disorders associated with ageing, and modulating ageing (e.g. age-modulating methods and uses), including in vivo, are also provided, said methods comprising modulating the expression and / or activity of the gene / combination of genes identified herein.

Provided herein are dosing regimens and methods for treating conditions and diseases characterized by a reduced expression or function of NaV1.1 protein by modulating splicing of a pre-mRNA encoded by an SCN1A gene. The dosing regimens and methods can be used to treat Dravet Syndrome or other conditions and diseases.

Disclosed herein are effector domains. The effector domains may be used with, for example, Cas proteins and CRISPR-Cas systems. The effectors may be used in combination with a Cas protein to form a fusion protein. The effectors may also be used in combination with an antibody that binds to a peptide epitope, wherein the peptide epitope is fused to a Cas protein. The compositions and methods comprising the effectors may be used to modulate gene expression.

The present invention provides an agent capable of modulating variable splicing in the intron of the SLC20A2 gene, and its use for modulating the expression of functional PiT2 protein to prevent and / or treat disease. The invention further provides a brain calcification model based on variable splicing in the intron of the human SLC20A2 gene, a preparation method of the brain calcification model, and application of the model to indication and analysis of the incidence stage of brain calcification, or to a model for researching phosphorus homeostasis imbalance in brain or a target of brain calcification, or to screening of drugs.

Disclosed are systems, components of systems, and methods for sensing extracellular ligands and / or modulating expression of an endogenous or exogenous gene in a cell. In some embodiments, the disclosed systems and methods comprise or utilize first and second exogenous extracellular sensors and / or nucleic acid sequences encoding the first and second exogenous extracellular sensors, wherein the first exogenous extracellular sensor comprises: a) a ligand binding domain; b) a transmembrane domain; c) a protease cleavage site; and d) a functional domain comprising an RNA-binding subdomain fused to a transcription regulatory subdomain; and the second exogenous extracellular sensor comprises: e) a ligand binding domain, f) a transmembrane domain, and g) a protease domain that cleaves the protease cleavage site of the first exogenous extracellular receptor.

The invention discloses a rape anti-freezing regulation gene and application, the rape anti-freezing regulation gene comprises BnRLCK1, BnRLCK2 and BnRLCK3 genes, mutants are generated from the BnRLCK1, BnRLCK2 and BnRLCK3 genes through a gene editing means, the sequences of the BnRLCK1, BnRLCK2 and BnRLCK3 genes are as shown in SEQ ID N0.1, SEQ ID N0.2 and SEQ ID N0.3, the rape anti-freezing regulation gene is prepared from Bnrlck1 / 2 / 3 mutants through gene editing, and the Bnrlck1 / 2 / 3 mutants are obtained through gene editing. The sequence of the brassica napus antifreeze regulatory gene after being edited is shown as SEQ ID N0.4, SEQ ID N0.5 and SEQ ID N0.6, and a three-target sgRNA sequence is designed aiming at the genes BnRLCK1, BnRLCK2 and BnRLCK3. According to the invention, new germplasm resources of the three homologous gene knockout mutants bnrlck1 / 2 / 3 of the brassica napus BnRLCK1, BnRLCK3 and BnRLCK3 of the brassica napus are obtained through a gene editing technology, and the germplasm resources are provided for cultivating new varieties of the anti-freezing brassica napus.

The present application provides materials and methods for treating a patient with one or more conditions associated with SCN9A whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and / or modulating the expression of SCN9A gene in a cell by genome editing.

Compositions are provided for transient transcriptional modulation of genes through delivery of RNA molecules encoding site-specific DNA binding molecules linked to transcriptional modulation domains, as well as methods for treating or preventing a disease or disorder, including a cancer, infectious disease, or immunological disease.

Provided herein are systems of modulating gene expression, methods of use thereof, and cells engineered thereof for the purpose of differentiating cells, for example beta cells.

To provide an agent and a composition for modulating, e.g., enhancing or reducing the expression of a hepatocyte nuclear factor 4-alpha (HNF4α) gene by targeting the HNF4α expression control region, as well as a use method thereof for treating an HNF4α-associated disease, e.g., cirrhosis.SOLUTION: The HNF4α gene may be in a cell, e.g., a mammalian cell, such as a mammalian somatic cell, e.g., a human somatic cell. The present invention also provides methods of using the agents and compositions of the invention for modulating the expression of an HNF4α gene or for treating a subject who would benefit from modulating the expression of an HNF4α gene, e.g., a subject suffering or prone to suffering from an HNF4α-associated disease.SELECTED DRAWING: None

To provide artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types.SOLUTION: An artificial expression construct comprises: (i) a concatemer of a specific sequence; (ii) a promoter; and (iii) a heterologous encoding sequence. The heterologous encoding sequence encodes an ion transporter, enzyme, transcription factor, receptor, membrane protein, cellular trafficking protein, signaling molecule, neurotransmitter, calcium reporter, channel rhodopsin, or CRISPR / CAS molecule etc., associated with a capsid that crosses the blood brain barrier. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in excitatory cortical neurons, such as primarily within cortical layers 2 / 3, 4, 5, and 6 and including those with extratelencephalic (ET) projections, intratelencephalic (IT) projections, and pyramidal tract (PT) projections.SELECTED DRAWING: None

Described herein are small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded by genes, pharmaceutical compositions comprising the same, and methods of use of the small molecule splicing modulator compounds for modulating splicing and treating diseases and conditions.

The invention relates to the technical field of gene engineering, in particular to a recombinant plasmid, a construction method and application of the recombinant plasmid to improvement of the synthesis capacity of nostoc flagelliforme pseudocladodin. The recombinant plasmid comprises a vector plasmid, and an sgRNA target sequence, an HR homologous sequence, a psbA promoter sequence and an ScyR regulator gene which are connected to the vector plasmid. The recombinant plasmid can over-express the key regulator gene at the MAAs gene cluster site of the nostoc flagelliforme, so that the pseudocladostin synthesis capability of the nostoc flagelliforme is remarkably improved. The recombinant plasmid is transformed into nostoc flagelliforme to obtain a nostoc flagelliforme mutant, and after the nostoc flagelliforme mutant is cultured for 15 days, the content of cladosporin is 2.4 times that of wild nostoc flagelliforme.

The p300 inhibitor specifically regulates the expression of genes that induce fibrosis, thereby acting only on fibrotic tissues and reducing the risk of adverse effects on normal tissues. Because the inhibitor blocks multiple fibrotic signaling pathways, it may be effective for fibrosis in various organs such as the liver, lungs, and kidneys. In addition, by modulating gene expression, the inhibitor has the potential to address the fundamental cause of the fibrotic process. The present invention is characterized in that bronopol is first identified as a specific example of such a p300 inhibitor. In particular, bronopol is an FDA-approved drug with established safety, and therefore is expected to reduce the time and cost required for conducting clinical trials.

The application of a gene BvMRS2-3 in improving plant salt tolerance, and belongs to the technical field of biological genes, and particularly relates to the application of a gene BvMRS2-3 in improving plant salt tolerance.The salt stress positive regulation gene BvMRS2-3, and the CDS sequence of the gene BvMRS2-3 is shown as SEQ ID NO.1.The protein encoded by the salt stress positive regulation gene BvMRS2-3, and the amino acid sequence of the protein is shown as SEQ ID NO.2.The application of the salt stress positive regulation gene BvMRS2-3 in improving plant salt tolerance.The BvMRS2-3 gene in the BvMRS2-3 gene heterologous overexpression plant prepared by the BvMRS2-3 gene can be stably inherited, wherein the heterologously expressed BvMRS2-3 gene can alleviate the inhibition of salt stress on the germination of the plant, promote the growth of the root length of the BvMRS2-3 heterologous overexpression plant, promote the Mg 2+ absorption and maintain the ion homeostasis in cells, which indicates that the BvMRS2-3 gene positively regulates the salt tolerance of the plant.

Compositions and methods for modifying genomic DNA sequences are provided. The methods produce double-stranded breaks (DSBs) at pre-determined target sites in a genomic DNA sequence, resulting in mutation, insertion, and / or deletion of DNA sequences at the target site(s) in a genome. Compositions comprise DNA constructs comprising nucleotide sequences that encode a Cms1 protein operably linked to a promoter that is operable in the cells of interest. The DNA constructs can be used to direct the modification of genomic DNA at pre-determined genomic loci. Methods to use these DNA constructs to modify genomic DNA sequences are described herein. Additionally, compositions and methods for modulating the expression of genes are provided. Compositions comprise DNA constructs comprising a promoter that is operable in the cells of interest operably linked to nucleotide sequences that encode a mutated Cms1 protein with an abolished ability to produce DSBs, optionally linked to a domain that regulates transcriptional activity. The methods can be used to up- or down-regulate the expression of genes at predetermined genomic loci.

The invention relates to a gene PagBES1 for regulating and controlling poplar wood yield and wood fiber length and application of the gene PagBES1, and belongs to the technical field of plant genetic engineering. The nucleotide sequence is shown as a sequence 3 in a sequence table, and the amino acid sequence is shown as a sequence 4 in the sequence table. The PagBES1 gene is transferred into the poplar, compared with a wild type, the plant height of the PagBES1 activated and expressed transgenic poplar is remarkably increased, the ground stem is remarkably thickened, the number of cell layers of stem vascular tissues is remarkably increased, the length and width of wood fibers are remarkably increased, secondary cell walls are remarkably deposited and thickened, and the phenotype of the PagBES1 inhibited and expressed transgenic poplar is opposite to the phenotype of the PagBES1 inhibited and expressed transgenic poplar. Therefore, the PagBES1 gene is a key regulatory gene for regulating the generation of poplar wood, and has important application value in the fields of forest gene engineering and clonal forestry.

The present disclosure provides an engineered nucleic acid sequence comprising a gene cassette, the gene cassette comprising one or more regulator genes, the regulator genes including an iadR gene, and at least three catabolic pathway genes downstream from the one or more regulator genes, the at least three catabolic pathway genes including an iadC gene, an iadD gene, and an iadE gene, wherein the engineered nucleic acid sequence is free of an iadA gene and iadB gene. The disclosure also provides bacteria cells comprising the engineered nucleic acid sequence, wherein the bacteria cell is capable of degrading indole-3-acetic acid. Methods for degrading auxin, controlling auxin, and promoting plant growth and root development are provided, comprising providing the bacteria cell and allowing the bacteria cell to interact with roots of a plant.

Described herein are small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded by genes, pharmaceutical compositions comprising the same, and methods of the small molecule splicing modulator compounds for modulating splicing and treating disease and conditions. In one aspect, disclosed herein are small molecule splicing modulators having a structure of Formula (II), or pharmaceutically acceptable salts thereof.

Articles, compositions, kits, and methods relating to nanostructures, including synthetic nanostructures, are provided. Certain embodiments described herein include structures having a core-shell type arrangement; for instance, a nanostructure core may be surrounded by a shell including a material, such as a lipid bilayer, and may include other components such as oligonucleotides. In some embodiments, the structures, when introduced into a subject, can be used to deliver nucleic acids and / or can regulate gene expression. Accordingly, the structures described herein may be used to diagnose, prevent, treat or manage certain diseases or bodily conditions. In some cases, the structures are both a therapeutic agent and a diagnostic agent.

The invention provides a gene for regulating and controlling the shape of a tomato leaf and application thereof, and a construction method of a recombinant expression vector is characterized in that an SlPUB26 gene is inserted into a vector containing a strong promoter by utilizing an overexpression transgenic technology. Transgenic plants with different gene expression level up-regulation multiples are obtained through agrobacterium tumefaciens-mediated transformation, so that single plants with different leaf phenotype change degrees are obtained. According to the method, a precise targeting sequence does not need to be designed, the miss-target hidden danger does not exist, and single plants with different SlPUB26 gene expression levels can be obtained only by detecting the gene expression quantity, so that the single plants with different leaf shape change degrees are obtained. The technical operation difficulty is low, and the method is more suitable for non-professional molecular breeding teams or grass-roots agricultural scientific research institutions. The higher the up-regulation multiple of the expression level of the SlPUB26 gene is, the lower the length-width ratio of the tomato leaf is, so that the shape of the tomato leaf can be quickly and accurately adjusted by adjusting the expression level of the SlPUB26 gene.

Genetic modulators comprising two or more artificial transcription factors for use in specific and active modulation of gene expression are provided.

Described herein are systems and methods for modulating gene expression. Also described herein are systems and methods for treating a disease or a condition by modulating gene expression.

The present invention discloses a tobacco calcium ion sensor-related gene and its application. The gene is named tobacco NtCBL4 gene, and its nucleotide sequence is: the nucleotide sequence shown in SEQ ID NO.1; a nucleotide sequence having the same function as the nucleotide sequence shown in SEQ ID NO.1 by substitution, deletion, and / or addition of one or more nucleotides; and a nucleotide sequence that hybridizes with the sequence shown in SEQ ID NO.1 under stringent conditions, wherein the stringent conditions are hybridization in 0.1×SSPE containing 0.1% SDS or 0.1×SSC containing 0.1% SDS at 65°C, and the membrane is washed with the solution. The present invention can control the salt tolerance of tobacco by regulating the expression level of the NtCBL4 gene, and obtain transgenic crops with different NtCBL4 gene expression levels and different salt tolerances through genetic engineering. Overexpression of NtCBL4 can reduce tobacco's tolerance to high salt.

Described herein are small molecule splice modulator compounds that modulate the splice of gene-encoded mRNA (e.g., pre-mRNA), pharmaceutical compositions comprising the same, and methods of using the small molecule splice modulator compounds to modulate splice and treat diseases and conditions.

Some embodiments of the present disclosure relate to one or more compositions for regulating the production of an antibody-like protein (ALP) and ribonucleic acid (RNA). In particular, the present disclosure relates to compositions for regulating the gene expression and, therefore, the production, of an ALP and an interfering RNA (iRNA), which may suppress viral infections. In some embodiments of the present disclosure, the target viral infection is a respiratory syncytial virus (RSV) infection.

The invention discloses streptomyces hygroscopicus ascomycetes subspecies and an obtaining method thereof. Regulating genes fkbN, fkbR1 and fkbR2 on an ascomycin biosynthesis gene cluster of the streptomyces hygroscopicus ascomycetes subspecies ATCC 14891 and a gene fkbS participating in synthesis of an ascomycin precursor ethyl malonyl coenzyme A are subjected to biosynthesis to obtain the streptomyces hygroscopicus ascomycetes subspecies. And carrying out overexpression on a regulatory gene SH03686 of a leucine response regulatory protein family (Lrp) outside the gene cluster, and screening an ascomycin high-yield strain. According to the invention, after combined overexpression of genes fkbS and fkbN and overexpression of a gene fkbR2 are carried out by a strain SN9 and a strain R2-17 in starting bacteria, the transcriptional level of regulatory genes (fkbN, fkbR2) on an ascomycin biosynthesis gene cluster and related genes participating in synthesis of precursors ethyl malonyl coenzyme A (fkbS, fkbE), piperidine acid (fkbL) and DHCHC (fkbO) are improved, and finally, the ascomycin yield is improved.