Abstract: Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C9ORF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).

Abstract: Disclosed herein are compositions and methods for reducing expression of C9ORF72 antisense transcript in an animal with C9ORF72 antisense transcript specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 antisense transcript specific inhibitors include antisense compounds.

Abstract: Disclosed herein are methods for treating/and or preventing diabetes using a specific inhibitor of SMAD7 expression or function. Also disclosed are methods of promoting organ and/or cell, e.g., pancreatic islet cell, survival after transplantation using a specific inhibitor of SMAD7 expression or function.

Abstract: The invention provides oligonucleotides complementary to a non-coding chimeric mitochondrial RNA as well as compositions and kits comprising the same, and their use in treating and preventing metastasis or relapse of a cancer in an individual previously treated for cancer with a therapy. The invention also provides oligonucleotides complementary to a non-coding chimeric mitochondrial RNA as well as compositions and kits comprising the same, and their use in treating a refractory cancer (e.g., a refractory HPV-associated cancer).

Abstract: The. invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with an antisense molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such antisense molecule used in said method.

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a Serum Amyloid A (SAA) gene, and methods of using the dsRNA to inhibit expression of SAA

Abstract: In certain embodiments, the present disclosure provides methods comprising contacting a cell with a compound comprising a modified oligonucleotide complementary to a nucleic acid transcript. In certain such embodiments, the modified oligonucleotide does not interact or interacts poorly with a mRNP complex or granule. In certain such embodiments the modifications and/or motifs of the modified oligonucleotide do not promote interaction with a mRNP complex or granule. In certain embodiments, the present disclosure provides methods comprising contacting a cell with a compound comprising a modified oligonucleotide thereby reducing the size or amount of protein aggregation in the cell. In certain such embodiments, the protein aggregate is a mRNP granule. In certain such embodiments, the modifications and/or motifs of the modified oligonucleotide promote interaction with a protein aggregate, such as a mRNP granule, that results in disruption of the protein aggregate.

Abstract: The inventions relate to compositions and articles of manufacture comprising connexin modulators, pannexin modulators, gap junction modulators, hemichannel modulators, and pannexin channel modulators and their use, alone or in combination, in treating ocular and other disorders.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) targeting a PROC gene, and methods of using the dsRNA to inhibit expression of PROC.

Abstract: Disclosed are double stranded RNA (dsRNA) molecules that are toxic to insect pests. In particular, interfering RNA molecules capable of modulating expression of a pest insect target gene and that are toxic to the insect pest are provided. Further, methods of making and using the interfering RNA, for example as the active ingredient in an insecticidal composition or in a transgenic plant, to confer protection from insect damage are disclosed.

Abstract: The present invention relates to a multi-conjugate of small interfering RNA (siRNA) and a preparing method of the same, more precisely a multi-conjugate of siRNA prepared by direct binding of double stranded sense/antisense siRNA monomers or indirect covalent bonding mediated by a cross-linking agent or a polymer, and a preparing method of the same. The preparing method of a siRNA multi-conjugate of the present invention is characterized by simple and efficient reaction and thereby the prepared siRNA multi-conjugate of the present invention has high molecular weight multiple times the conventional siRNA, so that it has high negative charge density, suggesting that it has excellent ionic interaction with a cationic gene carrier and high gene delivery efficiency.

Abstract: The present invention provides an oligonucleotide comprising the sequence 5?-GGAACAGTTCGTCCATGGC-3? (SEQ ID NO:2) for use in the treatment of inflammatory bowel disease in a human subject, wherein individual doses of from 100 mg to 350 mg of said oligonucleotide are administered to the subject on at least four separate occasions, wherein the separate occasions are each a week apart.

Abstract: The present invention relates to an isolated antisense oligonucleotide for use in a method of preventing or treating an inflammatory disease or condition of the buccal cavity in a canine subject, wherein said antisense oligonucleotide is directed against canine ICAM-1. The present invention further relates to compositions or articles of manufacture comprising said antisense oligonucleotide.

Abstract: A transaminase mutant and use hereof, the amino acid sequence of the transaminase mutant is an amino acid sequence in which the amino acid sequence as represented by SEQ ID NO: 1 is mutated, the mutated amino acid position being one or more selected from among F89, K193, P243, V234, I262, Q280, V379, R416, A417 and C418. The enzymatic activity and/or stability of the transaminase mutant is improved.

Abstract: The invention provides genetically engineered Wood-Ljungdahl microorganisms comprising one or more disrupted genes to strategically divert carbon flux away from nonessential or undesirable products and towards products of interest. The expression strategies of the invention enable the production of useful fuels and chemicals from gaseous substrates, such as carbon monoxide, carbon dioxide, and/or hydrogen.

Abstract: Provided are native promoters comprising polynucleotides isolated from Corynebacterium glutamicum, and mutant promoters derived therefrom, which may be used to regulate, i.e., either increase or decrease, on-pathway and/or off-pathway gene expression. Also provided are promoter ladders comprising a plurality of the promoters having incrementally increasing promoter activity. Also provided are host cells and recombinant vectors comprising the promoters, and methods of expressing ancillary genes of interest and producing biomolecules using the host cells.

Abstract: Reducing or eliminating activity of certain leucine-rich repeat, receptor-like kinases (LRR-RLK) polypeptides in a plant results in an increase in immune response in the plant, thereby conferring increased disease resistance in the plant. The genes that encode the polypeptides are in the same phylogenetic clade, and are termed Broad-Range Resistance (BRR) genes. The related LRR-RLK receptors are called Broad-Range Resistance (BRR) receptors.

Abstract: The present disclosure provides approaches for reducing nicotine. Also provided are tobacco plants with decreased tobacco-specific nitrosamines (TSNAs). The present disclosure further provides modified tobacco plants with low nicotine and increased antioxidant capacity. Further provided are tobacco plants or material with low nicotine and low TSNAs. Also provided is cured tobacco material of the tobacco plants provided herein and tobacco products comprising this cured tobacco material.

Abstract: This invention relates to crop plants of which the fruit dehiscence properties are modulated. More specifically the invention relates to improved methods and means for reducing seed shattering, or delaying seed shattering until after harvest, while maintaining at the same time an agronomically relevant treshability of the pods, and for increasing yield.

Abstract: The present disclosure provides methods for increasing drought resistance, salt resistance, photosynthetic rate, biomass production and water-use efficiency of a plant. The methods encompass expression of CAM-specific a phosphoenolpyruvate carboxylase (PEPC) in the plant. In comparison to a plant not manipulated in this manner, the disclosed, genetically-modified, plants display improved drought resistance and salt resistance. Also provided are plants that can be obtained by the method according to the invention, and nucleic acid vectors to be used in the described methods.

Abstract: Isolated polynucleotides and polypeptides encoded thereby are described, together with the use of those products for making transgenic plants with increased tolerance to abiotic stress (e.g., high or low temperature, drought, flood).

Abstract: Methods and materials for modulating cold tolerance levels in plants are disclosed. For example, nucleic acids encoding cold tolerance-modulating polypeptides are disclosed as well as methods for using such nucleic acids to transform plant cells. Also disclosed are plants having increased cold tolerance levels and plant products produced from plants having increased cold tolerance levels.

Abstract: Methods and materials for modulating cold tolerance levels in plants are disclosed. For example, nucleic acids encoding cold tolerance-modulating polypeptides are disclosed as well as methods for using such nucleic acids to transform plant cells. Also disclosed are plants having increased cold tolerance levels and plant products produced from plants having increased cold tolerance levels.

Abstract: In the present invention, HPPD polypeptides and plants containing them showing a full tolerance against one or more HPPD inhibitor herbicides belonging to various chemical classes are described. A set of mutant HPPD polypeptides have been designed which have either no or only a significantly reduced affinity to HPPD inhibitor herbicides and, at the same time, the rate of dissociation of the HPPD inhibitors of the mutant HPPD polypeptide is increased to such an extent that the HPPD inhibitors no longer act as slow-binding or slow, tight-binding inhibitors but, instead of this, have become fully reversible inhibitors. In particular, isolated polynucleotides encoding mutant HPPD polypeptides conferring tolerance to HPPD inhibitor herbicides belonging to various chemical classes are provided. Additionally, amino acid sequences corresponding to the polynucleotides are encompassed.

Abstract: This invention relates to the field of therapeutics. Most specifically, the invention provides methods of generating conditionally expressing vectors for one or more immunomodulators under the control of a gene expression modulation system in the presence of activating ligand and uses for therapeutic purposes in animals. These vector may be provided to treat a variety of disorders, e.g., neoplastic disorders, through direct injection or through in vitro engineered cells, such as dendritic cells.

Abstract: Mice comprising a modified p21-activated kinase (Pak) inhibitor domain (PID*), optionally linked with GST and capable of constitutive expression of PID are provided. Also provided are cells, tissue, and organs obtainable from such mice, and methods for producing mice comprising a modified p21-activated kinase (Pak) inhibitor domain (PID*).

Abstract: Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5? inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3? ITR.

Abstract: The present invention relates to an adenoviral vector comprising a regulatable Major Late Promoter and an exogenous transgene. The invention also provides cells comprising such adenoviral vectors, and processes using such vectors.

Abstract: Provided are methods for preparing T cells for cell therapy, compositions produced by the methods, and methods of administering the cells to subjects. In particular, the disclosure relates to preparation of engineered T cells, such as those expressing genetically engineered receptors, such as genetically engineered antigen receptors such as engineered (recombinant) TCRs and chimeric antigen receptors (CARs), or other recombinant chimeric receptors. Features of the methods include producing a more consistent and/or predictable T cell product and/or lower toxicity compared with other methods. The provided methods include incubating cells under stimulating conditions to induce expansion or proliferation of naive-like T cells compared to non-naive like T cells in the stimulated composition, which in turn can result in preferential transduction of cells derived from the naive-like T cells.

Abstract: The present disclosure provides expression constructs comprising a GLA transgene encoding the at least one ?-Gal A protein for use in expressing ?-Gal A proteins and preventing, inhibiting or treating Fabry disease or one or more symptoms associated with Fabry disease.

Abstract: The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).

Abstract: A method for carbon resource utilization is disclosed. According to one embodiment, the method includes (a) supplying carbon dioxide to a medium to form bicarbonate ions (HCO3?) (S100), (b) inoculating one or more microalgal species into the medium, followed by photo-culture (S200), and (c) supplying calcium ions (Ca2+) to the medium, where the microalgal species are photo-cultured, to produce calcite (CaCO3)-containing biomass (S300).

Abstract: The invention relates to a recombinant yeast comprising a recombinant yeast comprising a nucleotide sequence coding for a glycerol dehydrogenase, a nucleotide sequence coding for a ribulose-1,5-biphosphate carboxylase oxygenase (EC 4.1.1.39); a nucleotide sequence coding for a phosphoribulokinasey (EC 2.7.1.19); a nucleotide sequence allowing the expression of a glucoamylase (EC 3.2.1.20 or 3.2.1.3); and optionally a nucleotide sequence coding for a glycerol transporter. This cell can be used for the production of ethanol and advantageously produces little or no glycerol.

Abstract: Provided herein are Metschnikowia species that produce xylitol from xylose when cultured, as well as methods to make and use these Metschnikowia species.

Abstract: A method and cell line for producing polyketides in yeast. The method applies, and the cell line includes, a yeast cell transformed with a polyketide synthase coding sequence. The polyketide synthase enzyme catalyzes synthesis of olivetol or methyl-olivetol, and may include Dictyostelium discoideum polyketide synthase (“DiPKS”). Wild type DiPKS produces methyl-olivetol only. DiPKS may be modified to produce olivetol only or a mixture of both olivetol and methyl-olivetol. The yeast cell may be modified to include a phosphopantethienyl transferase for increased activity of DiPKS. The yeast cell may be modified to mitigate mitochondrial acetaldehyde catabolism for increasing malonyl-CoA available for synthesizing olivetol or methyl-olivetol.

Abstract: The present invention relates to a method for preparing an adipate ester or thioester. The invention further relates to a method for preparing adipic acid from said ester or thioester. Further the invention provides a number of methods for preparing an intermediate for said ester or thioester. Further the invention relates to a method for preparing 6-amino caproic acid (6-ACA), a method for preparing 5-formyl valeric acid (5-FVA), and a method for preparing caprolactam. Further, the invention relates to a host cell for use in a method according to the invention.

Abstract: The present invention provides a process for obtaining n+a oxidation and reduction products from a mixture of n sugars selected from the group consisting of C5 and C6 sugars, wherein n is at least 2 and a is at least 1, wherein at least two of the sugars in the mixture are present at a non-equimolar ratio to each other, wherein, in a first stage, at least one of the sugars which are present at a non-equimolar ratio to each other is oxidized enzymatically and, at the same time, at least one of the other sugars present at a non-equimolar ratio to each other is reduced enzymatically, and wherein, in the first stage, a portion of at least one of the sugars present at a non-equimolar ratio to each other is not converted, and which is characterized in that, in at least a second stage, at least a portion of the sugar not converted in the first stage is oxidized enzymatically by half and, respectively, is reduced enzymatically by the remaining half.

Abstract: The invention relates to a long-chain dibasic acid with low content of monobasic acid impurity and a production method thereof, in particular to the preparation of a long-chain dibasic acid producing strain by means of directed evolution and homologous recombination, and to the production of a long-chain dibasic acid with low content of monobasic acid impurity by fermentation of said strain. The invention relates to a mutated CYP52A12 gene, homologous gene or variant thereof, which, relative to GenBank Accession Number AY230498 and taking the first base upstream of the start codon ATG as ?1, comprises a mutation. The invention relates to a strain comprising said mutated CYP52A12 gene, homologous gene or variant thereof wherein when the strain is fermented to produce a long-chain dibasic acid, the content of monobasic acid impurity in the fermentation product is significantly reduced.

Abstract: A method for producing an L-amino acid such as L-glutamic acid is provided. An L-amino acid is produced by culturing in a medium a bacterium having an L-amino acid-producing ability, which has been modified so that the activity of a c1795 protein is reduced or the activity of a protein of which the expression is repressed by a c1795 protein is increased, and collecting the L-amino acid from the medium or cells of the bacterium.

Abstract: Disclosed are methods for chemoenzymatic synthesis of S-Nucleosyl Amino acid probes (SNA), analogs of S-adeno-syl-L-methionine and S-adenosyl-L-homo-cysteine, analogs synthesized by the methods, and uses thereof.

Abstract: The present invention is directed to a system and method for producing a vitamin B12 enriched Duckweed Bacterial Culture (DBC) composition. The present invention further discloses a composition comprising a vitamin B12 enriched DBC, wherein said composition comprises at least one Lemnoideae species and at least one vitamin B12 producing bacteria species, further wherein said vitamin B12 content in said composition is in the range of between about 0.01 and about 100 ?g per 100 g of said DBC.

Abstract: The present invention relates to the use of limited amounts of cysteine and tryptophan in the cell culture medium during production of recombinant proteins, and in particular antibodies. Proteins and antibodies produced under such controlled conditions exhibit reduced heterogeneity, in particular reduced charge variants heterogeneity.

Abstract: The present invention is related to a novel enzymatic process for production of retinyl esters, such as in particular retinyl long chain esters, via conversion of retinol, which process includes the use of enzymes having acyltransferase activity. Said process might be used for biotechnological production of vitamin A.

Abstract: The present invention is related to production of retinyl esters, such as in particular retinyl acetate, an important building block for production of vitamin A. The retinyl esters might be generated via enzymatic conversion of retinol which process includes the use of enzymes with acetyl-transferase (ATF) activity, thus acetylating retinol into retinol/retinyl acetate. Said process is particularly useful for biotechnological production of vitamin A.

Abstract: Embodiments disclose apparatus, methods and software for performing biological screening and analysis implemented using an instrument platform capable of detecting a wide variety of cell-based secretions, expressed proteins, and other cellular components. The platform may be configured for simultaneous multiplexed detection of a plurality biological components such that a large number of discrete samples may be individually sequestered and evaluated to detect or identify constituents from the samples in a highly parallelized and scalable manner.

Abstract: A cyanuric acid-responsive biosensor includes a host strain or cell-free system including a first and a second expression cassette; the first expression cassette including, in operable communication, a constitutive promoter and a gene encoding an AtzR cyanuric acid-responsive transcription factor; and the second expression cassette including, in operable communication, a promoter regulated by the cyanuric acid-responsive transcription factor and a gene encoding a reporter protein, wherein the first expression cassette expresses the AtzR cyanuric acid-responsive transcription factor, and, in the presence of cyanuric acid, the AtzR cyanuric acid-responsive transcription factor drives expression of the reporter protein.

Abstract: A method for detecting the presence of bacterial spores is described by measuring microbial metabolic activity over time. Spores are distinguished from vegetative cells and other microorganisms by detecting a burst of metabolic activity indicating germination of spores. This method may be used to detect bacterial spores in a commercial process system, such as a papermaking system within the time frame of a typical work shift.

Abstract: Methods of detecting biologicals in samples is provided herein. The detection is based on the formation of aggregates. The disclosed compositions include labeling particles and/or aggregating particles. The labeling particles and the aggregating particles may each include a receptor bound to the particle. The receptor can be either directly attached to the particle or indirectly attached to the particle through a linker. One method of detection may be visual and another may include advanced quantification of the formed aggregates.

Abstract: Methodologies to detect off-target mutations induced by the deaminase activity of Base Editing technology.

Abstract: Methods, compositions, and kits for stabilizing both human and microbial deoxyribonucleic acid (DNA) present in complex biological samples, such as feces, are disclosed. In particular, aqueous compositions for stabilizing DNA contained in biological samples at ambient temperature are disclosed, together with associated methods and kits using same. In one aspect, the compositions comprise a chelating agent present at a concentration of at least about 150 mM, and the composition has a pH of at least about 9.5.