Abstract: The present invention relates to an antagonist of the Two-Pore Domain Potassium Channel (TASK-1) K2P3.1 for use in the prevention and/or treatment of cardiac arrhythmia in a subject. The invention also relates to a nucleic acid molecule usable in the prevention and/or treatment of cardiac arrhythmia in a subject. The invention further relates to a cell comprising said nucleic acid molecule. The invention further relates to a vector comprising said nucleic acid molecule.

Abstract: The present invention relates to a composition for genome editing using a CRISPR/Cpf1 system and a use thereof and, more particularly, to a composition for genome editing comprising: a CRISPR RNA (crRNA) including a guide sequence capable of hybridizing with a target nucleotide sequence, and a uridine repeat sequence connected to the 3?-end of the guide sequence, or a DNA encoding the same; and a Cpf1 protein or a DNA encoding the same, a method for genome editing using the same, a method for construction of a genetically modified organism, and a genetically modified organism. The present invention can increase an indel efficiency and decrease off-target activity in genome editing of eukaryotic cells using the CRIPSPR/Cpf1 system and thus can easily construct a genetically modified cell or genetically modified animal or plant having a desired gene inserted thereinto (knock-in) or deleted therefrom (knock-out).

Abstract: This disclosure relates to novel huntingtin targets. Novel oligonucleotides for the treatment of Huntington's disease are also provided.

Abstract: Methods and compositions capable of modulating activity of TLX (NR2E1), a nuclear receptor essential for neural stem cell self-renewal are provided. The modulation may comprise downregulating TLX expression and/or modulating TET3. In addition, methods of delivering shRNAs using dendrimer nanoparticles into glioblastoma stem cells are provided. The methods and compositions are useful for treating and preventing the progression of brain cancer, e.g., glioblastoma.

Abstract: Improved compositions and methods for treating a disease or disorder through target exon skipping, and preferably muscular dystrophy by administering antisense thiomorpholino molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping to produce a functional Dystrophin protein.

Abstract: The present invention relates to exosomes, loaded with genetic material and methods of producing them and to the use of such exosomes for delivering genetic material in vivo, in particular the use of such exosomes in methods of gene therapy or gene silencing.

Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the HMGB 1 gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a HMGB1 gene and to methods of N preventing and treating an HMGB1-associated disorder, e.g., metabolic disorder or non-alcholic fatty liver disease, e.g., non-alcoholic steatohepatitis (NASH).

Abstract: The present disclosure relates to an inhibitor of Dynamin 2 for use in the treatment of centronuclear myopathies. The present disclosure relates to pharmaceutical compositions containing Dynamin 2 inhibitor and to their use for the treatment of centronuclear myopathies. It also deals with a method for identifying or screening molecules useful in the treatment of a centronuclear myopathy.

Abstract: Compositions and methods of genome engineering in vitro and in vivo are provided. In some embodiments, the compositions are triplex forming molecules that bind or hybridize to a target region sequence in the human cystic fibrosis transmembrane conductance regulator (CFTR) gene. Preferably the triplex forming molecules are peptide nucleic acids that include a Hoogsteen binding peptide nucleic acid (PNA) segment and a Watson-Crick binding PNA segment collectively totaling no more than 50 nucleobases in length, wherein the two segments can binid or hybridize to a target region in the CFTR gene having a polypurine sequences and induce strand invasion, displacement, and formation of a triple-stranded molecule among the two PNA segments and the target region's sequence. Methods of using the triplex forming molecules to treat cystic fibrosis are also provided.

Abstract: Provided are methods of assessing activity of a promoter region. The methods include culturing a cell including a nucleic acid, the nucleic acid including a region that encodes an enzyme donor (ED) operably coupled to a promoter region, under conditions in which the ED is expressed when the promoter region is active. The methods further include contacting the ED, if expressed, with an enzyme acceptor (EA) to form ED-EA complexes having enzymatic activity. The methods further include detecting the level of the enzymatic activity to assess activity of the promoter region. Activity of the promoter region may be indicative, and therefore may be used to assess, the activity of a cellular signaling pathway of interest and/or of endogenous or exogenous (e.g., introduced) transcription factors of interest. Cells, compositions, and kits that find use, e.g., in practicing the methods of the present disclosure, are also provided.

Abstract: Methods of controlling bacteria cells are disclosed. These methods comprise upregulating expression of a DVU2956 sigma 54-dependent enhancer-binding protein (EBP) in bacteria cells, resulting in (i) dispersing a biofilm of the cells or reducing biofilm formation by the cells, and/or (ii) reducing hydrogen sulfide formation by the cells. Further disclosed are methods of identifying compounds for controlling bacteria cells as in (i) and/or (ii) above. Polynucleotides and cells are disclosed that can optionally be used to practice compound identification methods.

Abstract: In accordance with the invention, isolated nucleic acids, expression methods, host cells, expression vectors, and DNA constructs for producing proteins, and proteins produced using the expression methods are described. More particularly, nucleic acids isolated from Pichia pastoris wherein the nucleic acids have promoter activity are described. The invention also relates to expression methods, host cells, expression vectors, and DNA constructs, for using the Pichia pastoris promoters to produce proteins, and to the proteins produced using the expression methods.

Abstract: Methods and systems are provided for generating and utilizing a bacterial composition that comprises at least one genetically engineered bacterial strain that fixes atmospheric nitrogen in an agricultural system that has been fertilized with more than 20 lbs of Nitrogen per acre.

Abstract: Methods are provided to mutate, in a targeted manner, the genome of a plant cell using a double stranded DNA break inducing enzyme. Also provided are plants, in particular Brassica plants that yield seeds producing oils having a reduced total saturated fatty acid content, and method for making such plants.

Abstract: This document provides oilseed plants (e.g., pennycress plants) having increased levels of one or more saturated fatty acids, increased levels of one or more polyunsaturated fatty acids (PUFAs), altered (e.g., increased or decreased) levels of oleic acid, and/or altered (e.g., increased or decreased) levels of erucic acid. For example, oilseed plants having reduced polypeptide levels and/or reduced polypeptide activity of one or more polypeptides involved in triglyceride synthesis (e.g., diacylglycerol O-acyltransferase 1 (TAG1) can have increased levels of stearic acid, increased levels of one or more PUFAs, altered levels of oleic acid, and/or altered levels of erucic acid. Also provided herein are methods and materials for making and using oilseed plants having increased levels of one or more saturated fatty acids, increased levels of one or more PUFAs, altered levels of oleic acid, and/or altered levels of erucic acid.

Abstract: Compositions and methods for improving plant growth are provided herein. Polynucleotides encoding ADP-glucose pyrophosphorylase small subunit (AGPaseSS) proteins, polypeptides encompassing AGPaseSS proteins, and expression constructs for expressing genes of interest whose expression may improve agronomic properties including but not limited to crop yield, biotic and abiotic stress tolerance, and early vigor, plants comprising the polynucleotides, polypeptides, and expression constructs, and methods of producing transgenic plants are also provided.

Abstract: The disclosure provides nucleic acids, and variants and fragments thereof, derived from strains of Bacillus thuringiensis encoding variant polypeptides having increased pesticidal activity against insect pests, including Lepidoptera and Coleopteran. Particular embodiments of the disclosure provide isolated nucleic acids encoding pesticidal proteins, pesticidal compositions, DNA constructs, and transformed microorganisms and plants comprising a nucleic acid of the embodiments. These compositions find use in methods for controlling pests, especially plant pests.

Abstract: A method of altering a eukaryotic cell is provided including transfecting the eukaryotic cell with a nucleic acid encoding RNA complementary to genomic DNA of the eukaryotic cell, transfecting the eukaryotic cell with a nucleic acid encoding an enzyme that interacts with the RNA and cleaves the genomic DNA in a site specific manner, wherein the cell expresses the RNA and the enzyme, the RNA binds to complementary genomic DNA and the enzyme cleaves the genomic DNA in a site specific manner.

Abstract: The present invention provides a kit of vectors for transducing an immune cell with multiple transgenes comprising: (i) a first vector which comprises a first transgene and a nucleotide sequence encoding a transcription factor and; and (ii) a second vector which comprises a second transgene wherein expression of the second transgene within a host cell is dependent upon expression of the transcription factor.

Abstract: The present invention relates to a cell line, use of the cell line and a method for producing infectious viral particles using said cell line.

Abstract: Disclosed herein are viral vectors for use in recombinant molecular biology techniques. In particular, the present disclosure relates to self-limiting viral vectors comprising genes encoding site-specific endonucleases as well as recognition sequences for site-specific endonucleases such that expression of the endonuclease in a cell cleaves the viral vector and limits its persistence time. In some embodiments, the viral vectors disclosed herein also carry directives to delete, insert, or change a target sequence.

Abstract: The present disclosure relates to compositions and methods for introducing an mRNA into stem cells, such as HSPCs, and for delivering gene editing components to such cells in vitro. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 complex in HSPCs, and methods and delivery systems for achieving such gene modification in HSPCs.

Abstract: Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.

Abstract: The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.

Abstract: The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.

Abstract: The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.

Abstract: The present invention relates to S53 proteases and to the use of S53 protease in processes for converting starch to ethanol.

Abstract: A process of producing methacrylic acid and/or derivatives thereof including the following steps: (a) biologically converting isobutyryl-CoA into methacrylyl-CoA by the action of an oxidase; and (b) converting methacrylyl-CoA into methacrylic acid and/or derivatives thereof. The invention also extends to microorganisms adapted to conduct the steps of the process.

Abstract: The disclosure provides ?-ketoisocaproic acid decarboxylase and ?-keto-3-methylvaleric acid decarboxylase and recombinant microorganisms that host these enzymes. Methods involve the use of recombinant microorganisms to increase the production of isoamyl alcohols, their corresponding acids and their derivatives from carbon sources.

Abstract: A CO2, bioconversion process includes providing a CO2 containing substrate to a bioreactor, the CO2 containing substrate including about 5 to about 90 mole % CO2; and fermenting the CO2 containing substrate with an acetogenic bacteria carrying a sodium translocating ATPase. The medium including less than about 0.01 grams per liter yeast extract, less than about 0.01 grams per liter carbohydrate, a sodium ion concentration provided by a sodium ion feed rate of about 290 to about 8750 ?g/gram of cells/minute, and a pH of about 4 to about 6.9.

Abstract: Provided is a method of producing one or more sesquiterpene compounds comprising: contacting an acyclic FPP precursor with a polypeptide having terpene synthase activity, wherein the polypeptide comprises an amino acid sequence that has at least 55% sequence identity to SEQ ID NO: 1, to produce one or more terpenes selected from the group consisting of isovalencene, spirovetiva-1(10),7(11)-diene and valencene or derivatives thereof, or mixture of sesquiterpenes comprising one or more of isovalencene, spirovetiva-1(10),7(11)-diene and/or valencene; and optionally isolating the one or more terpenes or the mixture.

Abstract: A recombinant cell for producing a fatty acid ester. The recombinant cell is genetically engineered to produce a reduction in free fatty acids compared to a cell that has not been similarly genetically engineered. Methods for producing fatty acid esters while decreasing free fatty acid production are also described.

Abstract: The present invention relates to a method for decontaminating glucose polymers or the hydrolysates of the pro-inflammatory molecules thereof. Said method includes a) providing glucose polymers or the hydrolysates thereof, b) optionally, detecting or assaying the pro-inflammatory molecules in the glucose polymers or the hydrolysates thereof provided in Step a), and c) carrying out the following purifying steps: i. treatment using an enzymatic preparation having detergent properties and clarification properties; ii. treatment using a pharmaceutical-grade activated carbon with very high adsorption properties and “micropore” porosity; iii. optionally, treatment using a second activated carbon with “mesopore” porosity; iv. passing them over a macroporous adsorbent polymer resin having porosity greater than 100 Angstroms; and v. continuous ultrafiltration at 5 kDa.

Abstract: This Invention discloses a method for production of N-Acetyl-D-Glucosamine and/or D-Glucosamine Salt by microbial fermentation. The method is intended to manufacture N-Acetyl-D-Glucosamine and/or D-Glucosamine Salt in higher efficiency and higher yield, by increasing the effects of N-Acetyl-D-Mannosamine Kinase.

Abstract: A method of producing steviol glycoside compositions and the use thereof in foods, beverages and other consumables, is described.

Abstract: Methods for recombinant production of steviol glycoside and compositions containing steviol glycosides are provided by this invention.

Abstract: One aspect of the invention provides a system for drug discovery, drug development, drug screening, or drug validation. The system includes: a sample chamber comprising a target protein and a drug candidate that may interfere with the target protein in the sample chamber, wherein the sample chamber is configured to: detect one or more of the following: (a) interference between the drug candidate the target protein and/or (b) one or more dynamics of the drug candidate on the target protein, wherein the one or more dynamics comprise affinity of the drug candidate to the target protein, and select the drug candidate if one or more desirable dynamics is detected. The system includes one or more immobilized surfaces and is configured to detect interactions between the drug candidate and the target protein at the single-molecule level.

Abstract: The invention relates to a gene construct for detecting the presence of microorganisms that produce chemical molecules of the Acyl-homoserine lactone (AHL) type, comprising a first expression cassette that includes a copper-inducible promoter operably linked to the gene encoding the RhlR protein and, downstream of said first expression cassette, a second expression cassette including a promoter that is induced by the AHL-RhlR complex, said promoter being operably linked to a gene encoding a reporter protein. The invention also includes a genetically modified biosensor cell to detect the presence of microorganisms comprising said gene construct, as well as the method of detecting the presence of microorganisms that produce AHL-type chemical molecules by immobilizing the biosensor in an organic matrix, generating a surface that can be used to expose a sample of a fluid such as air or a liquid medium, and thus detect these molecules mediated by the biosensor cell.

Abstract: Provided herein are methods of using atomic force microscopy (AFM) to measure the adhesion force between a cell surface target and a ligand (e.g., an antibody) that binds to the cell surface target. Such adhesion force serves as an in vitro metric for predicting the in vivo tumor recognition and/or anti-tumor efficacy of antibody-directed nanomedicine.

Abstract: The present invention provides a method for evaluating tumorigenicity of a test cell, including transplanting the cell into anterior chamber of an eye of an experimental animal, and observing the presence or absence of tumor formation. According to the present invention, tumorigenicity can be evaluated in a short period and conveniently.

Abstract: Methods of detecting actively growing host organisms, including bacterial organisms, involving the detection of ribonucleic acid (RNA) products produced by unmodified infectious bioagents that infect the host organisms are provided. Related methods of assessing drug susceptibility and resistance are further provided. In addition to these surrogate detection methods, related reaction mixtures, kits, systems, and microfluidic cards are also provided.

Abstract: The present invention addresses the problem of providing a novel method for classifying cancer cells by an analysis method in which measurement of the activity of two types of protein kinase is used. Cancer cells are newly classified and drug sensitivity is predicted on the basis of the ratio of the activity of two types of protein kinase derived from the same sample.

Abstract: A method for the detection of impaired responsiveness of CD4+ T-cells to regulatory T-cells (Treg), referred to as Treg resistance. The method includes measuring the expression levels of phosphatase and tension homolog (PTEN) in activated CD4+ T-cells. Furthermore, a screening method for the detection of an autoimmune disease or a condition, may comprise the steps of generating a functional gene expression profile by measuring the expression levels of phosphatase and tension homolog (PTEN) in Treg-resistant CD4+ T-cells from patients suffering of an autoimmune disease or condition, and comparing the obtained gene expression profile with the expression profile from Treg-sensitive CD4+ T-cells from healthy controls. PTEN can be utilized in a screening system for the detection of impaired responsiveness of CD4+ T-cells to Treg.

Abstract: The present disclosure provides nucleic acid-based nanoswitch catenanes and methods of use. A nanoswitch catenane may include a single-stranded nucleic acid comprising a first and second terminal domain linked to each other to form a host ring by one of a first, second or third switchable bridges, wherein the first switchable bridge is formed in the presence of a reaction agent through the reaction of two cognate functional groups, each linked to a terminal domain of the single-stranded nucleic acid, wherein the second switchable bridge is formed in the presence of a biomolecule of interest through binding of the bio-molecule of interest to two cognate antibodies, each linked to a terminal domain of the single stranded nucleic acid, and wherein the third switchable bridge is a link between two cognate functional groups that breaks in the presence of a dissociation agent. A nanoswitch catenane may also include a circular nucleic acid guest ring catenated with the host ring.

Abstract: In order to perform relative quantification of gene expression in a cell/tissue repeatedly, it is required to supply a large number of standards. By employing an absolutely quantified standard including a synthetic DNA plasmid or the like, the present invention stably provides uniform standards for the repeated gene expression analysis over a long period of time. Further, the present invention enables stable execution of repeated gene expression analysis with high reproducibility by using such a standard. In addition, the present invention provides a method for stably controlling the quality of a cell or tissue over a long period of time by using the method, and also provides a standard used for the method.

Abstract: Disclosed herein are methods of extracting genetic material from a diverse population of one or more types of microbes in a sample. Microbes can be prokaryotes or eukaryotes and may include bacteria, archaea, fungi, protozoa, helminths, parasites, viruses, phages, and others. Extraction may be from a single sample and subsequent identification may be through a molecular method such as qPCR, PCR, RFLP, SSCP, allele specific PCR, targeted sequencing, pull down sequencing, whole shotgun sequencing, or other methods. Also provided are methods that include extracting nucleic acid molecules from a variety of organisms such as fungi (i.e., Saccharomyces spp.), animal cells (Bos taurus), plants (e.g., Hordeum vulgare) from the gut of a human subject, performing a metagenomics analysis therefrom, and determining a probiotic treatment or dietary guidance for the subject based on the metagenomics analysis.

Abstract: Provided is a primer pair of primers for methicillin-resistant gene detection for the purpose of achieving highly sensitive methicillin-resistant gene detection. Said primer pair comprises a combination of SEQ ID NO: 3 and SEQ ID NO: 7, a combination of SEQ ID NO: 2 and SEQ ID NO: 9, a combination of SEQ ID NO: 1 and SEQ ID NO: 8, a combination of SEQ ID NO: 1 and SEQ ID NO: 9, a combination of SEQ ID NO: 4 and SEQ ID NO: 11, a combination of SEQ ID NO: 5 and SEQ ID NO: 12, a combination of SEQ ID NO: 6 and SEQ ID NO: 10, or a combination of SEQ ID NO: 6 and SEQ ID NO: 12.

Abstract: The disclosure provides an asymmetric PCR amplification method for preparation of single-stranded product and primers and kits useful therefor.

Abstract: A method for processing a nucleic acid, in which the nucleic acid is exposed to an aqueous medium which includes a polyol in sufficient proportion for at least a portion of the nucleic acid to enter or remain in an extra-solution phase. Thus, a polyol may be used to bind a nucleic acid which is in solution to a solid support or to wash a nucleic acid on a solid support whilst maintaining it on the support. The polyol may for example be a C2-C10 alkanediol.

Abstract: Disclosed is a stable double-stranded nucleic acid signal probe in which single-stranded nucleic acids constituting a double-stranded nucleic acid are complementarily crosslinked (or covalently bonded) with each other, and thus are not affected by temperature, pH, salinity, ionic strength, etc. Also disclosed is a method capable of detecting a target molecule by forming a complex of the stable double-stranded nucleic acid signal probe and the target molecule using the signal probe in the detection of the target molecule, separating only the signal probe from the complex by heating or the like, and detecting the signal probe.