Abstract: Disclosed herein are methods of treating a tumor in a subject, including administering to the subject one or more miRNA nucleic acids or variants (such as mimics or mimetics) thereof with altered expression in the tumor. Also disclosed herein are compositions including one or more miRNA nucleic acids. In some examples, the miRNA nucleic acids are modified miRNAs, for example, and miRNA nucleic acid including one or more modified nucleotides and/or a 5?-end and/or 3?-end modification. In particular examples, the modified miRNA nucleic acid is an miR-30a nucleic acid. Further disclosed herein are methods of diagnosing a subject as having a tumor with altered expression of one or more miRNA nucleic acids. In some embodiments, the methods include detecting expression of one or more miRNAs in a sample from the subject and comparing the expression in the sample from the subject to a control.

Abstract: The present disclosure relates to modified antisense oligonucleotides. The nucleotides described herein are of 10 to 40 nucleobases and include a targeting sequence complementary to a target region within intron 1 of a pre-mRNA of the human alpha glucosidase (GAA) gene. The target region includes at least one additional nucleobase compared to the targeting sequence, wherein the at least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional nucleobase is internal to the target region.

Abstract: Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.

Abstract: Methods, kits and devices for dosing a subject to reduce a hypersensitivy response to a lipid-formulated nucleic acid (e.g., RNA) molecule are disclosed.

Abstract: The invention relates to a RNA interference triggers for inhibiting the expression of an AAT gene through the mechanism of RNA interference. The invention also relates to a pharmaceutical composition comprising the AAT RNAi trigger together with an excipient capable of improving delivery of the RNAi trigger to a liver cell in vivo. Delivery of the AAT RNAi trigger to liver cells in vivo provides for inhibition of AAT gene expression and treatment of alpha 1-antitrypsin deficiency and associated diseases.

Abstract: Products and associated methods are described for the delivery of one or more small interfering RNAs (siRNAs) to an avian cell using a nonpathogenic bacterium for the prevention or treatment of Avian Influenza Virus (AIV). The siRNA is complementary to an mRNA of the AIV NP or PA sequence. In challenge studies with chickens, the siRNA is shown to prevent the onset of clinical symptoms or reduce the severity of disease, including reducing viral titers or virus shedding.

Abstract: The objective of the present invention is to provide a DNA aptamer that can specifically binding to a molecular targeted medicine, a composition comprising the DNA aptamer, and a method for detecting a molecular targeted medicine using the DNA aptamer. A DNA aptamer comprising specifically binding to a molecular targeted medicine, a composition for detecting a molecular targeted medicine comprising the DNA aptamer, a kid for detecting a molecular targeted medicine, and a method for detecting a molecular targeted medicine comprising using the DNA aptamer.

Abstract: The present invention provides compositions and methods for treating cancer with peptide nucleic acid agents. In some embodiments, the present invention provides methods and compositions relating to peptide nucleic acid agents that target oncogenes. For example, the present invention provides compositions, including pharmaceutical compositions, comprising agents specific for BRAF V600E inhibition, or fragments or characteristic portions thereof. The present invention further provides various therapeutic and/or diagnostic methods of using BRAF V600E specific peptide nucleic acid agents and/or compositions.

Abstract: The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as ?-thalassemia or hemochromatosis.

Abstract: Methods of treating a wound in a subject are provided comprising administering to the subject an amount of an inhibitor of Fidgetin-like 2. Compositions and pharmaceutical compositions comprising an amount of an inhibitor of Fidgetin-like 2 are also provided. Methods are also provided for identifying an inhibitor of Fidgetin-like 2.

Abstract: Nonnaturally occurring host cells altered to increase their ability to transfer genetic molecules into the host cells as compared to an unaltered host cell are provided. Also provided are methods for identifying endogenous loci of a host cell which inhibit transformation efficiency and/or electroporation of genetic molecules into the cell as well as methods for producing nonnaturally occurring host cells with enhanced transformation efficiency and/or the modified ability to allow for genomic integration of an exogenous DNA sequence via electroporation. Methods for producing biochemicals and products produced with the nonnaturally occurring host cells are also provided. In particular, a host Cupriavidus necator cell is provided in which a chromosomal gene encoding a restriction endonuclease has been disrupted, leading to an increase in transformation efficiency.

Abstract: A method for cloning an exon into a plasmid vector. Exons related to prostate cancer (PVT1 exon 9, PVT1 exon 4A or PVT1 exon 4B) and miRNAs (miR-1205 or miR-1207-3p) are transformed into the plasmid vector. The cloned exons or miRNAs are linearized and their concentrations quantified. Serial dilutions in conjunction with spectroscopy permit the construction of a standard curve that permits absolute quantification of the exons or miRNAs in a biological sample from a patient.

Abstract: Described herein are methods and compositions relating to male-sterile wheat, as well as uses thereof and methods for propagating and maintaining the same.

Abstract: The present invention relates to methods of producing industrial products from plant lipids, particularly from vegetative parts of plants. In particular, the present invention provides oil products such as biofuel, and processes for producing these products, as well as plants having an increased level medium chain fatty acids such as lauric acid and myristic acid. In one particular embodiment, the present invention relates to combinations of modifications in a fatty acid thioesterase and one or more acyltransferases. In an embodiment, the present invention relates to a process for extracting lipids. In another embodiment, the lipid is converted to one or more hydrocarbon products in harvested plant vegetative parts to produce alkyl esters of the fatty acids which are suitable for use as a renewable biofuel.

Abstract: A method of making an antibody in plants that binds to ricin is described. The method comprises (a) introducing a nucleic acid molecule encoding a heavy chain variable region of the antibody and a nucleic acid molecule encoding a light chain variable region of the antibody into a plant or plant cell; and (b) growing the plant or plant cell to obtain a plant that expresses the antibody or antibody fragment. The disclosure also relates to anti-ricin antibodies and antibodies fragments as well as methods of using same in therapy and prophylaxis.

Abstract: A method of regulating plant stomata conductance is provided. The method comprises modulating in the plant the level and/or activity of a type B hexokinase in a guard cell specific manner, thereby regulating plant conductance.

Abstract: The invention relates to nucleic acid molecules which impart resistance to rhizomania, in particular to Beet Necrotic Yellow Vein Virus (BNYVV) in a plant, in particular of the genus Beta, and to plants containing such nucleic acid molecules. The invention further relates to methods for producing such BNYVV resistant plants and to marker-based methods for identifying and selecting BNYVV resistant plants, as well as to methods for controlling infection with the pathogen BNYVV.

Abstract: Compositions having pesticidal activity and methods for their use are provided. Compositions include isolated and recombinant polypeptides having pesticidal activity, recombinant and synthetic nucleic acid molecules encoding the polypeptides, DNA constructs and vectors comprising the nucleic acid molecules, host cells comprising the vectors, and antibodies to the polypeptides. Nucleotide sequences encoding the polypeptides can be used in DNA constructs or expression cassettes for transformation and expression in organisms of interest. The compositions and methods provided are useful for producing organisms with enhanced pest resistance or tolerance. Transgenic plants and seeds comprising a nucleotide sequence that encodes a pesticidal protein of the invention are also provided. Such plants are resistant to insects and other pests. Methods are provided for producing the various polypeptides disclosed herein, and for using those polypeptides for controlling or killing a pest.

Abstract: Non-human animal cells and non-human animals comprising CRISPR/Cas synergistic activation mediator system components and methods of making and using such non-human animal cells and non-human animals are provided. Methods are provided for using such non-human animals to increase expression of target genes in vivo and to assess CRISPR/Cas synergistic activation mediator systems for the ability to increase expression of target genes in vivo.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells to provide gene therapy for pyruvate kinase deficiency.

Abstract: The present invention provides new stable packaging cell lines and producer cell lines as well as methods to obtain them, and a new method to produce lentiviral vectors using such cell lines. New methods and packaging cell lines of the invention are generated using a baculo-AAV hybrid system for stable expression of structural and regulatory lentiviral proteins, such system comprising a baculoviral backbone containing an integration cassette flanked by AAV ITR, in combination with a plasmid encoding rep protein. This system allows to obtain a stable integration of the structural and regulatory HIV-1 proteins gag/pol and rev. The system allows to obtain a first intermediate including only the structural and regulatory HIV proteins gag/pol and rev, to be used as starting point to obtain stable packaging cell lines as well as producer cell lines.

Abstract: Expression of a transgene is driven in a host cell using a pol I promoter which is not endogenous to an organism from the same taxonomic order from which the host cell is derived.

Abstract: Provided are methods for producing recombinant adeno-associated virus (rAAV) vector particles at high recovery or high titer. Also provided are methods that concentrate rAAV vectors to a high concentration, for example, up to 5E+13 (5×1013) vector genomes per milliliter (Vg/ml) with little if any rAAV aggregates.

Abstract: The present invention relates to the field of (vector) vaccines, and especially to novel EHV's having an inactivation of UL18 and/or UL8. The present invention further concerns related expression cassettes and vectors, which are suitable to express genes of interest, especially antigen encoding sequences. The viral vectors of the present invention are useful for producing an immunogenic composition or vaccine.

Abstract: The present invention includes the Paramyxovirus Parainfluenza Virus 5 (PIV5) as an oncolytic agent for treating various cancers, including, but not limited to breast cancer, lung cancer and melanoma. PIV5 oncolytic agents include both wild type PIV5 and various recombinant PIV5 constructs. Recombinant PIV5 constructs may include PIV5 lacking the conserved C-terminus of the V protein (PIV5V?C), PIV5 with mutations in the N-terminus of the V/P protein (PIV5CPI?), and PIV5 expressing MDA-7/IL-24 (rPIV5-MDA7), rPIV5-V/P-CPI?, rPIV5-CPI+, rPIV5-Rev, rPIV5-RL, rPIV5-P-S157A, rPIV5-P-S308A, rPIV5-L-A1981D, rPIV5-F-S443P, rPIV5-MDA7, rPIV5?SH-CPI?, or rPIV5?SH-Rev. Also included are methods of making and using such oncolytic agents and compositions including such oncolytic agents.

Abstract: In an illustrative embodiment, automated multi-module cell editing instruments comprising one or more flow-through electroporation devices or modules are provided to automate genome editing in live cells.

Abstract: The present invention provides a preparation method for an anti-porcine reproductive and respiratory syndrome cloned pig, comprising: transferring CRISPR/Cas9 targeting vectors and CD163 gene homologous recombination modification vectors into fibroblasts of a pig to obtain positive clone cells, a seventh exon of porcine endogenous CD163 gene being replaced with a eleventh exon of human CD163-L1 gene, so that the positive clone cells are incapable of mediating invasions of PRRSV; taking the positive cell as nuclear transfer donor cells and oocytes as nuclear transfer recipient cells to obtain a cloned embryo by adopting a somatic cell nuclear transfer technology; and impregenating a pig by transferring the cloned embryo into the uterus of the pig, to obtain a cloned pig.

Abstract: A technique for editing genome using a single strand polynucleotide consisting of: (i) (ia) a modified nucleotide sequence having a deletion of an arbitrary endogenous nucleotide sequence from an arbitrary nucleotide sequence in genomic DNA; (ib) a modified nucleotide sequence having an insertion of an arbitrary nucleotide sequence to an arbitrary nucleotide sequence in genomic DNA; or (ic) a modified nucleotide sequence having a substitution of an arbitrary endogenous nucleotide sequence with another nucleotide sequence in an arbitrary nucleotide sequence in genomic DNA; or (ii) a nucleotide sequence having 90% or more identity with the modified nucleotide sequence (except the same nucleotide sequence as the sense strand nucleotide sequence).

Abstract: The present invention provides homologous recombination vectors to insert transgenic DNA in cells. These vectors shorten the production time and allow for easy generation of genetically modified cells. The invention allows the user to test multiple tags and to generate homozygous modified cell line using the homologous recombination vector. The invention can be used to generate knockout cells, to generate cell lines with knockin genes, to generate cell lines for drug screening against any target, to create transgenic animals, or in gene therapy.

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 a two-stage process for production of drop-in fuels/alcohols (methanol, ethanol or butanol) from volatile fatty acids produced either synthetically from fossil resources or as metabolic intermediates in acidification step of anaerobic digestion process from waste biomass and organic materials.

Abstract: A process for the preparation of bio-based malonic acid and crystalline calcium malonate is provided. The calcium malonate is highly pure and provides a source of malonic acid made from a renewable carbon source rather than existing processes which rely on the use of petroleum-based products. The calcium malonate provides an improved source of malonic acid, which is important to many industrial processes.

Abstract: Provided are a filamentous fungus mutant strain having an effect of improving C4 dicarboxylic acid productivity, and a method for producing C4 dicarboxylic acid using the filamentous fungus mutant strain. The method for producing a C4 dicarboxylic acid comprises culturing a filamentous fungus mutant strain which has enhanced expression of a polypeptide consisting of the amino acid sequence represented by SEQ ID NO: 2 or an amino acid sequence having at least 90% identity thereto and having catalase activity is enhanced.

Abstract: The present invention relates to a process for the production of methyl methacrylate. The process of the present invention comprises the steps of: a) providing a microorganism in a fermentation medium, under conditions which said microorganism will produce a C3-C12 methacrylate ester; b) providing an organic phase in contact with the fermentation medium, said organic phase including C3-C12 methacrylate ester in a higher concentration than that in the fermentation medium; c) removing organic phase containing the said C3-C12 methacrylate ester from contact with the fermentation medium; and d) transesterifying the removed C3-C12 methacrylate ester with methanol, optionally after separation from the organic phase, to produce methyl methacrylate.

Abstract: Mutant thioesterases having enhanced medium chain substrate activity, polynucleotides encoding and configured to express the mutant thioesterases in a transformed host cell, host cells transformed to contain the polynucleotides, and methods of using same.

Abstract: Provided is a method of activating gene expression using a protein having 90% or more sequence identity to SEQ ID NO:45. The protein activates the expression of a gene upon induction with a medium-chain or long-chain alkane or a medium-chain or long-chain fatty acid methyl ester. Also provided is a whole-cell catalytic system regulated by a medium-chain or long-chain alkane or a medium-chain or long-chain fatty acid methyl ester. The system includes a recombinant microbial cell expressing the protein and an alkane monooxygenase. Also provided is a method of preparing a medium-chain or long-chain alkane terminal oxidation product using the whole-cell catalytic system.

Abstract: The present invention generally relates to the field of biotechnology as it applies to the production of aryl sulfates using recombinant host cells. More particularly, the present invention pertains to recombinant host cells comprising (e.g., expressing) a polypeptide having aryl sulfotransferase activity, wherein said recombinant host cells have been modified to have an increased uptake of sulfate compared to identical host cells that does not carry said modification. Further provided are processes for the production of aryl sulfates, such as zosteric acid, employing such recombinant host cells.

Abstract: The present invention relates to an L-cysteine-producing mutant microorganism having introduced therein genes encoding enzymes which are involved in the L-cysteine metabolic pathway, and more particularly to an L-cysteine-producing mutant microorganism having introduced therein cysE, cysK and cysR, which are genes encoding enzymes which are involved in the L-cysteine metabolic pathway, and to a method of producing L-cysteine using the mutant microorganism. According to the present invention, L-cysteine can be produced with high efficiency as a result of regulating metabolic fluxes associated with the L-cysteine metabolic pathway of the mutant microorganism and regulating a system for supplying a sulfur source essential for synthesis of L-cysteine.

Abstract: The invention provides non-naturally occurring microbial organisms containing caprolactone pathways having at least one exogenous nucleic acid encoding a butadiene pathway enzyme expressed in a sufficient amount to produce caprolactone. The invention additionally provides methods of using such microbial organisms to produce caprolactone by culturing a non-naturally occurring microbial organism containing caprolactone pathways as described herein under conditions and for a sufficient period of time to produce caprolactone.

Abstract: An improved dry grind system and method for producing a sugar stream from grains or similar carbohydrate sources and/or residues, such as for biochemical production, with front end oil separation. Prior to or after saccharification, oil can be removed from a sugar/carbohydrate stream. After saccharification and prior to a sugar conversion process, the sugar/carbohydrate stream includes a desired Dextrose Equivalent (DE) where DE describes the degree of conversion of starch to dextrose can be produced, with such sugar stream being available for biochemical production, e.g., alcohol production, or other processes. In addition, the systems and methods also can involve the removal of certain grain components, e.g., corn kernel components, including protein and/or fiber. In other words, oil separation and sugar stream production occurs on the front end of the system and method.

Abstract: The application relates to an enzyme composition, a process for the preparation thereof and the use of the enzyme composition in enzymatic hydrolysis.

Abstract: The present disclosure relates to a novel polypeptide having an activity of exporting 5?-inosine monophosphate, a microorganism comprising the same, a method for preparing 5?-inosine monophosphate using the same, and a method for increasing export of 5?-inosine monophosphate.

Abstract: This disclosure describes a method of purifying several full-length oligonucleotide targets from corresponding synthesis truncation products, in a way that ensures roughly stoichiometric equality among the targets.

Abstract: In some embodiments, the disclosure relates generally to methods, as well as related compositions and kits for recombinase-mediated nucleic acid amplification, such as recombinase-polymerase amplification (RPA), of a nucleic acid template using at least one blocked primer that contains a 5? domain, at least one nucleotide that is cleavable by an RNase H enzyme, a 3? domain, wherein the primer is not extendable by a polymerase, and wherein the 3? domain has a length of 7-100 nucleotides, for example 10-30 nucleotides. These methods and the use of a blocked primer reduce or eliminate non-specific amplification products, such as primer dimers, which are generated in RPA reactions.

Abstract: A novel method for producing a mogrol glycoside and mogrol has been required. The present invention provides a method for producing a mogrol glycoside and/or mogrol, said method comprising a step for cleaving at least one glucoside bond in a mogrol glycoside.

Abstract: The invention relates to a method for producing a lysate used for cell-fee protein biosynthesis, comprising the following steps: a) a genomic sequence in an organism, which codes for an essential translation product that reduces the yield of cell-fee protein biosynthesis, is replaced by the foreign DNA located under a suitable regulatory element, said foreign DNA coding for the essential translation product that additionally contains a marker sequence; b) the organism cloned according to step a) is cultivated; c) the organisms from the culture obtained in step b) are lysed; and d) the essential translation product is eliminated by means of a separation process that is selective for the marker sequence. Also discussed are said lysate and the use thereof.

Abstract: Disclosed are methods, systems, components, and compositions for cell-free synthesis of glycosylated carrier proteins. The glycosylated carrier proteins may be utilized in vaccines, including anti-bacterial vaccines. The glycosylated carrier proteins may include a bacterial polysaccharide conjugated to a carrier, which may be utilized to generate an immune response in an immunized host against the polysaccharide conjugated to the carrier. The glycosylated carrier proteins may be synthesized in cell-free glycoprotein synthesis (CFGpS) systems using prokaryote cell lysates that are enriched in components for glycoprotein synthesis such as oligosaccharyltransferases (OSTs) and lipid-linked oligosaccharides (LLOs) including OSTs and LLOs associated with synthesis of bacterial O antigens.

Abstract: The present disclosure provides nanoparticle transducers and methods of use thereof for the detection of analyte concentrations in a fluid. Nanoparticle transducers can comprise a nanoparticle, such as a Pdot, coupled to an enzyme that catalyzes a reaction with the analyte. The nanoparticle transducers further comprise chromophores that emit fluorescence that varies as a function of the concentration of one of the elements of the reaction. The nanoparticle transducer thus changes fluorescence as the analyte concentration changes, transforming analyte concentration values into fluorescence intensities. The measurement of these intensities provides a measurement of the analyte concentration. The nanoparticle transducers are biocompatible, allowing for use in vivo, for the monitoring of analyte blood concentrations such as blood glucose concentrations.

Abstract: The present disclosure provides a system and method for depleting target nucleic acids from a nucleic acid sample. In one aspect, a kit according to the present disclosure includes a plurality of DNA probes. Each of the DNA probes is hybridizable to form a heteroduplex with at least one of a plurality of target RNA transcripts in a nucleic acid sample. The number of unique target RNA transcripts hybridized by the plurality of DNA probes is at least three. The kit further includes an enzyme having RNA-DNA hybrid ribonucleotidohydrolase activity, where degrades at least the RNA portion of the heteroduplex.

Abstract: The present invention provides for single-molecule profiling of combinatorial protein modifications and single-molecule profiling of combinatorial protein modifications combined with single-molecule sequencing of protein/nucleic acids complexes. High-throughput single-molecule imaging was applied to decode combinatorial modifications on millions of individual nucleosomes from pluripotent stem cells and lineage-committed cells. Applicants identified bivalent nucleosomes with concomitant repressive and activating marks, as well as other combinatorial modification states whose prevalence varies with developmental potency. Applying genetic and chemical perturbations of chromatin enzymes show a preferential affect on nucleosomes harboring specific modification states. The present invention also combines this proteomic platform with single-molecule DNA sequencing technology to simultaneously determine the modification states and genomic positions of individual nucleosomes.