Abstract: Provided are oligomeric compounds, methods, and pharmaceutical compositions for modulating expression of SCN1A RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a developmental or epileptic encephalopathic disease, such as, for example, Dravet Syndrome. Such symptoms include seizures, sudden unexpected death in epilepsy, status epilepticus, behavioral dysfunctions, movement and balance dysfunctions, orthopedic conditions, motor dysfunctions, cognitive impairment, delayed language and speech, visual motor integration dysfunctions, visual perception dysfunctions, executive dysfunctions, and dysautonomia.

Abstract: Disclosed herein are compositions and methods for modulating, such as decreasing, the expression of a mutant histone 3.3 (H3.3) allele or a mutant H3.3 gene. In some embodiments, the compositions are antisense oligonucleotides (ASOs), such as gapmer ASOs and splice-modulating ASOs, that target a region of a mutant H3.3 allele or a mutant H3.3 gene, or products thereof, to reduce expression of the mutant H3.3 allele or a mutant H3.3 gene. The compositions and methods are useful in the treatment of diseases and disorders associated with a mutant H3.3 allele or a mutant H3.3 gene, such as cancer.

Abstract: Described herein are methods of modulating OTC gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for increasing expression of Ornithine transcarbamylase (OTC), thereby treating diseases associated with aberrant gene expression.

Abstract: The invention relates to a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, or exons 50-53 of the DMD pre-mRNA in a patient, the method comprising providing the patient with the molecule. The invention also relates to the molecule as such.

Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include Parkinson's disease.

Abstract: The disclosure provides biomarkers for diagnosis and monitoring of acute hepatic porphyria (AHP). The disclosure further provides methods for selection of agents for treatment of AHP using the biomarkers. The disclosure further provides kits for practicing the methods provided herein.

Abstract: The present invention provides double stranded ribonucleic acid (dsRNA) agents for inhibiting expression of a target gene, comprising an antisense strand which is complementary to the target gene; a sense strand which is complementary to the antisense strand and forms a double stranded region with the antisense strand; and one or more C22 hydrocarbon chains conjugated to one or more internal positions on at least one strand, compositions comprising such dsRNA agents, and methods of use thereof for treating a subject having a skeletal muscle disorder, a cardiac muscle disorder, or an adipose tissue disorder.

Abstract: Provided herein are synthetic RNAs comprising a piRNA sequence and a target sequence for a gene of interest. The synthetic RNAs find use in methods of gene editing, in particular in methods of silencing expression of the gene of interest.

Abstract: Therapeutic compounds for red blood cell-mediated delivery of an active pharmaceutical ingredient to a cancer cell are described. The therapeutic compounds are configured to bind CD47 on the surface of a red blood cell and to be subsequently transferred to CD47 on the surface of the cancer cell, the therapeutic compound ultimately being internalized by the cancer cell via endocytosis.

Abstract: The present invention introduces drug-loaded vehicles conjugated with aptamers, comprising DNA, RNA, or modified nucleic acid sequences designed to selectively and strongly bind to specific target sites on the eye, as well as binding to an ocular device such as a contact lens. Additionally, this innovative approach offers a targeted and sustained drug delivery method, addressing issues such as burst release of drugs, poor affinity between drug and material, and non-targeted drug delivery, while maintaining non-invasiveness and convenience.

Abstract: The present disclosure provides a Mycobacterium tuberculosis (Mtb) adenosine triphosphate (ATP) synthase-expressing recombinant bacterium, and a construction method and an expression method thereof, and belongs to the technical field of exogenous expression. The present disclosure provides a construction method of an Mtb ATP synthase-expressing recombinant bacterium, including the following steps: using a Mycobacterium smegmatis (Msm) competent cell containing an auxiliary gene knockout plasmid as a basal cell; transferring an Mtb ATP synthase gene cluster with an affinity purification tag into the basal cell, knocking out a Msm ATP synthase genome, and collecting a strain without the auxiliary gene knockout plasmid after conducting repeated subculture; and transferring the Mtb ATP synthase gene cluster into the strain by prokaryotic expression to obtain the Mtb ATP synthase-expressing recombinant bacterium.

Abstract: As disclosed herein, optimal native genomic loci of soybean plants have been identified that represent best sites for targeted insertion of exogenous sequences.

Abstract: The present disclosure relates to the field of plant molecular biology, more particularly to regulation of gene expression in plants.

Abstract: Disclosed are double stranded RNA molecules that are toxic to coleopteran insects. In particular, interfering RNA molecules capable of interfering with pest target genes and that are toxic to the target pest are provided. Further, methods of making and using the interfering RNA, for example in transgenic plants or as the active ingredient in a composition, to confer protection from insect damage are disclosed.

Abstract: The present invention relates to a tobacco plant, etc. This tobacco plant has either one or both of the following: (i) an endogenous gene containing, as an encoding region, a nucleic acid containing a base sequence encoding SEQ ID NO: 2 or an amino acid sequence having at least 95% identity with SEQ ID NO: 2, in which at least one codon corresponding to the amino acid sequence has mutated to a stop codon; and (ii) an endogenous gene containing, as an encoding region, a nucleic acid containing a base sequence encoding SEQ ID NO: 4 or an amino acid sequence having at least 95% identity with SEQ ID NO: 4, in which at least one codon corresponding to the amino acid sequence has mutated to a stop codon.

Abstract: The present invention relates to methods of synthesizing long-chain polyunsaturated fatty acids, especially eicosapentaenoic acid, docosapentaenoic acid and docosahexaenoic acid, in recombinant cells such as yeast or plant cells. Also provided are recombinant cells or plants which produce long-chain polyunsaturated fatty acids. Furthermore, the present invention relates to a group of new enzymes which possess desaturase or elongase activity that can be used in methods of synthesizing long-chain polyunsaturated fatty acids. In particular, the present invention provides ?3 destaurases, ?5 elongases and ?6 desaturases with novel activities. Also provided are methods and DNA constructs for transiently and/or stably transforming cells, particularly plant cells, with multiple genes.

Abstract: The present invention relates to methods and means for producing nitrogenase polypeptides in the mitochondria of plant cells. The present disclosure provides plant cells that express one or more MTP-Nif fusions and/or translational NifD-NifK and NifE-NifN fusions. The present disclosure also provides nucleic acid constructs encoding these fusions as well as expression constructs for expression and targeting of the fusions to the mitochondria of plant cells. The present disclosure also provides transgenic plants comprising the plant cells of the invention and products obtained therefrom.

Abstract: Described herein are modified plants, plant cells, and plant seeds that have at least one mutant loss-of-function JAZ gene and at least one loss-of-function cdk8 gene. Such plants are highly resistant to pests and environmental stress and have restored growth and increased seed yield compared to plant lines with a jazD genetic background.

Abstract: The present invention relates to the field of agriculture and more particularly to the use of attenuated ToBRFV mutants for cross-protecting Tm-22 resistant tomato varieties against ToBRFV infection and reducing disease symptoms. Also provided are methods of protecting agricultural crops such as tomatoes against said virus.

Abstract: Herein, is describe a new technology that exploits modified-virus-like particles (VLP) to improve deliverability and increase effectiveness of dsRNAs/chemicals/peptides to difficult-to-control phytophagous insect pests, including hemipteran and lepidopteran insects. This technology has also the potential to control plant pathogens including fungi, like Asian Soybean Rust (ASR).

Abstract: Compositions and methods for controlling pests are provided. The methods involve transforming organisms with a nucleic acid sequence encoding an insecticidal protein. In particular, the nucleic acid sequences are useful for preparing plants and microorganisms that possess insecticidal activity. Thus, transformed bacteria, plants, plant cells, plant tissues and seeds are provided. Compositions are insecticidal nucleic acids and proteins of bacterial species. The sequences find use in the construction of expression vectors for subsequent transformation into organisms of interest including plants, as probes for the isolation of other homologous (or partially homologous) genes. The pesticidal proteins find use in controlling, inhibiting growth or killing Lepidopteran, Coleopteran, Dipteran, fungal, Hemipteran and nematode pest populations and for producing compositions with insecticidal activity.

Abstract: Compositions and methods are provided for genome modification of a nucleotide sequence located in or near a male fertility gene of Ms1 or Ms5 in the genome of a plant cell or plant to produce a male-sterile plant. In some examples, the methods and compositions employ a guide RNA/Cas endonuclease system for modifying or altering target sites located in or near a male fertility gene of Ms1 or Ms5 in the genome of a plant cell, plant or seed to produce a male-sterile plant. Also provided are compositions and methods employing a guide polynucleotide/Cas endonuclease system for genome modification a nucleotide sequence located in or near a male fertility gene of Ms1 or Ms5 in the genome of a plant cell to produce a male-sterile plant. Compositions and methods are also provided for restoring fertility to a Ms1 or Ms5 nucleotide sequence to a male-sterile Ms1 or Ms5 plant.

Abstract: Provided herein are CRISPR/Cas-related methods and components for editing a target nucleic acid sequence in a HBG1 and/or HBG2 gene regulatory region, and applications thereof in connection with methods of increasing expression of fetal hemoglobin and treating ?-hemoglobinopathies including sickle cell disease and ?-thalassemia.

Abstract: The present disclosure relates to a polynucleotide or a set of polynucleotides comprising a first nucleic acid molecule encoding an innate immune inhibitor, e.g., an influenza non-structural (NS1) protein, and a second nucleic acid molecule encoding a heterologous target mRNA. The disclosure also includes methods of making the polynucleotides, methods of modifying a cell, and methods of treating a subject using the same.

Abstract: Described herein are compositions and methods of using stable cells lines in combination with transient transfection to produce recombinant AAV (rAAV).

Abstract: Provided herein are compositions and methods for use of self-inactivating recombinant vectors (SIRV) encoding Class 2 Type V and guide ribonucleic acid (gRNA) sequences useful for nucleic acid sequence editing, and including self-inactivating components. The SIRV may be delivered to cells as part of an AAV vector to target a gene of interest.

Abstract: The present application relates to an adeno-associated viral (AAV) vector, comprising a muscle cell-specific promoter and a nucleic acid molecule encoding an AU-rich mRNA binding factor 1 (AUF1) protein or a functional fragment thereof, where the nucleic acid molecule is heterologous to and operatively coupled to the muscle cell-specific promoter. Also disclosed are compositions comprising the AAV vector, as well as methods of promoting muscle regeneration in injured muscle, a method of treating degenerative skeletal muscle loss in a subject, methods of preventing traumatic muscle injury in a subject such as Duchenne Muscular Dystrophy, methods of treating traumatic muscle injury in a subject, and methods of treating muscle loss due to aging in a subject.

Abstract: Provided herein are prime editing methods and compositions for treatment of genetic disorders such as myotonic dystrophy.

Abstract: The present disclosure provides systems and methods for transposing a cargo nucleotide sequence to a target nucleic acid site. These systems and methods may comprise a first double-stranded nucleic acid comprising the cargo nucleotide sequence, wherein the cargo nucleotide sequence is configured to interact with a retrotransposase, and the retrotransposase, wherein said retrotransposase is configured to transpose the cargo nucleotide sequence to the target nucleic acid site.

Abstract: Methods and compositions for genetically altering induced pluripotent stem cells (iPSCs) to allow for efficient viral delivery to differentiated cell lines derived from this background.

Abstract: A method of making graphene transparent by fermentation contains: a step of preparing prerequisites; a first step in which the graphene mother bacteria, edible powders, powdered sugars, and a warm water and mixed and heated to produce a fermented graphene lump; a second step in which fermented graphene lump is smashed and smoothed out to a holding tray, then the holding tray is dried to form graphene enzyme lump. The method further contains: a third step in which the graphene enzyme lump is refined to graphene enzyme powders by a stirrer; a fourth step in which re-fermenting the graphene enzyme powders of the third step by repeating the first step, the second step and the third step; a fifth step in which the re-fermented graphene powders are soaked to produce edible powders, and the warm water is removed; a step sixth in which a transparent graphene containing probiotics is produced.

Abstract: A marine-derived nanoselenium-producing strain and a method for preparing nanoselenium by using the strain are provided in the present disclosure, belonging to the field of nano biomaterials. According to the present disclosure, a Bacillus strain capable of preparing nanoselenium is screened from sediments taken from the Arctic Ocean, named Bacillus sp. Q72, which has been deposit in the China General Microbiological Culture Collection Center, and the deposit number is CGMCC No.26355. The extracellular fluid of this strain is used to directly reduce high concentrations of sodium selenite to obtain selenium nanoparticles.

Abstract: Methods and systems that use fluorocarbons for designing scalable living, in-line extraction of heterologous metabolites from microbial cultures are disclosed. The disclosed systems and methods are organism agnostic and be applied to bacteria, yeasts, filamentous microbes, and algae. The disclosed methods and systems use pperfluorocarbon solvent to extract terpenes from engineered microorganisms and allows non-lethal milking of hydrocarbon products from filamentous and single-celled/multicelled microbial cultures. The disclosed process preferably does not use pressure gradients and relies on the physical interaction of microbes with fluorocarbon at satp and standard temperature of microbial cultivation to partition non-native products. The disclosed process and system also allow for increased production of products providing a physical sink which enables the forward reactions to produce more products than in its absence.

Abstract: Described are two-stage anaerobic digestion processes and systems that are coupled with post-hydrolysis ammonia removal for digestion of high-nitrogen feedstock into biogas such as biomethane. The processes and related systems comprise a first-stage anaerobic digestion unit to obtain hydrolyzed material; a NH3-stripping unit to obtain an ammonia-stripped slurry; and a second-stage anaerobic digestion unit for the methanogenesis of the ammonia-stripped slurry and production of a digestate and biogas. One process and system is adapted for operation in a continuous or semi-continuous mode. Another process and system comprise stripping with upgraded biogas.

Abstract: Some variations provide a process to produce renewable biogas and cellulosic fiber from a cellulose-rich feedstock, comprising: obtaining a cellulose-rich feedstock that is reduced in concentration of impurities and has a length less than 5 inches; feeding the cellulose-rich feedstock and water to a vacuum steam-explosion vessel, thereby generating a first cellulose-rich intermediate stream; feeding the cellulose-rich intermediate stream to a secondary separation vessel to remove remaining impurities, thereby generating a second cellulose-rich intermediate stream; feeding the second cellulose-rich intermediate stream to an enzymatic-hydrolysis vessel to convert the second cellulose-rich intermediate stream to low-viscosity cellulosic fibers comprising cellulose oligomers; feeding the low-viscosity cellulosic fibers to an anaerobic digester, thereby generating a biogas stream and residual fiber; optionally, recycling residual fiber to the enzymatic-hydrolysis vessel and/or to the anaerobic digester; and recove

Abstract: The present invention provides for a mechanism to reduce glycerol production and increase nitrogen utilization and ethanol production of recombinant microorganisms. One aspect of this invention relates to strains of S. cerevisiae with reduced glycerol productivity that get a kinetic benefit from higher nitrogen concentration without sacrificing ethanol yield. A second aspect of the invention relates to—metabolic modifications resulting in altered transport and/or intracellular metabolism of nitrogen sources present in corn mash.

Abstract: Processes for production of phloroglucinol or derivative thereof. An aspect of the present disclosure relates to an enzymatic process for production of phloroglucinol or derivative thereof from polyphenol. The present disclosure also relates to a process for production of phenolic derivatives. Accordingly, another aspect of the present disclosure relates to an enzymatic process for production of phenolic derivatives from polyphenol. The processes of the present disclosure affords facile and environment friendly production of phloroglucinol or derivative(s) thereof and/or phenolic derivative(s).

Abstract: The present invention provides for a genetically modified host cell comprising one or more enzymes described herein, wherein the genetically modified host cell is capable of producing malonate from methane.

Abstract: The present invention relates to the development of antibacterial biocellulosic patches/membranes for transdermal drug delivery. Novel high cellulose producing bacterial strain Komagatacibacter hansenii (MBS-8) has been isolated. The production and purification process of biocellulosic membranes is developed in such a way that the membranes produced are of desired thickness and length, suitable for drug impregnation in a cost-effective manner for the development of transdermal patches. Further, a process for efficient drug impregnation of Mupirocin in BC membranes has been developed. Weight and drug content of mupirocin impregnated BCM were found to be uniform.

Abstract: Methods for producing a linear deoxyribonucleic acid (DNA) product with enhanced resistance to nuclease digestion are provided.

Abstract: Methods of preparing highly purified steviol glycosides are described. The methods include utilizing enzyme preparations and recombinant microorganisms for converting various starting compositions to target steviol glycosides. The highly purified steviol glycosides are useful as non-caloric sweetener, flavor enhancer, sweetness enhancer, flavor stabilizer, flavoring with modifying properties (FMP), foaming suppressor and solubility enhancing agent in consumable products such as any food, beverages, pharmaceutical compositions, tobacco products, nutraceutical compositions, and oral hygiene compositions.

Abstract: The present invention relates to a method for producing collagen peptide preparations comprising recombinant collagen peptides, to collagen peptide preparations produced by means of said methods, to products containing the collagen peptide preparations and to uses of the aforementioned preparations and products.

Abstract: Solutions and methods are disclosed for the extraction and quantification of cellulose derived from cellulosic biomass samples. Improved extraction yields and selectivities are provided through the use of an extraction solvent mixture comprising an aprotic solvent such as DMSO, a quaternary ammonium salt such as TBAF, and an quaternary ammonium base such as TBAOH. The extracted cellulose can be optionally precipitated using disclosed precipitation solutions to further improve cellulose purity. Extracted cellulose can be measured by hydrolyzing the cellulose to glucose or cellobiose, or by using disclosed spectrophotometric assays of cellulose-salt complexes.

Abstract: Coagulation and fibrinolysis assays and related compositions, systems, methods, and kits are provided. In some embodiments, a coagulation and fibrinolysis assay may utilize one or more biological molecules. For instance, the assay may comprise combining a blood or blood-derived patient sample with the biological molecule(s) and measuring one or more properties of the sample associated with coagulation and/or fibrinolysis. The biological molecules may serve to shorten the assay duration and/or enhance the sensitivity of the assay relative to certain conventional assays. In certain embodiments, the biological molecules may allow pathological coagulation and/or fibrinolysis phenotypes to be elucidated. The coagulation and fibrinolysis assays described herein may be used for a wide variety of clinical and/or laboratory applications, including the diagnosis of certain coagulation and/or fibrinolysis disorders, such as trauma-induced coagulopathy and hyperfibrinolysis.

Abstract: The present disclosure provides methods and systems for generating and processing optically tagged beads. A nucleic acid probe comprising a fluorescent tag may be hybridized to a bead to generate an optically tagged bead. The nucleic acid probe hybridized to the optically tagged bead may be detectable by one or more signals and may be substantially unreactive to one or more chemical reactions performed with, or in the presence of, the optically tagged bead.

Abstract: Provided herein are methods, compositions, and kits for capturing a ligation product, in which the method can include covalently reacting a capture moiety of a capture probe with a reactive moiety of the ligation product to form a captured ligation product.

Abstract: Chromogenic conjugates for color-based detection of targets are described. The conjugates comprise a chromogenic moiety such as a rhodamine, rhodol or fluorescein. The chromogenic moiety is linked to a peroxidase substrate. The chromogenic conjugates can be used in immunohistochemical analysis and in situ hybridization. The conjugates can be used to detect 1, 2, 3 or more targets in a sample by color.

Abstract: Disclosed herein, inter alia, are compositions and methods of use thereof for interrogating a tissue sample.

Abstract: The invention is directed to devices and methods for performing rapid low-cost bioassays in self-contained disposable cartridges that provide efficient mixing of sample and reactants under a layer of liquid wax. Some embodiments additionally use gravity assisted distribution of sample and assay reagents in conjunction with an appliance containing all necessary valves, pneumatic sources, heat sources and detection stations.

Abstract: Methods for constructing consecutively connected copies of nucleic acid molecules are disclosed. Consecutively connected copies of nucleic acid molecules can be used to perform sequencing of the same nucleic acid molecules several times, improving overall accuracy of sequencing. Connected copies of nucleic acid molecules can be constructed by circularizing nucleic acid molecules, performing rolling circle amplification and debranching with nicking and polymerases comprising 5?-3? exonuclease and/or flap endonuclease activity.