Abstract: The present invention relates to microfluidic fluidic devices, methods and systems as microfluidic kidney on-chips, e.g. human Proximal Tubule-Kidney-Chip, Glomerulus (Kidney)-Chip, Collecting Duct (Kidney)-Chip. Devices, methods and systems are described for drug testing including drug transport and renal clearance. Further, such devices, methods and systems are used for determining drug-drug interactions and their effect upon renal transporter functions. Importantly, they may be used for pre-clinical and clinical drug development for treating kidney diseases and for personalized medicine.

Abstract: The instant technology relates to a production system to produce AAV vectors in a serum free suspension platform and at high titers. This technology uses reagents comprising media, cells, transfection reagent, AAV enhancer, and a lysis buffer, each of which is designed to provide maximal AAV production from suspension culture of mammalian cells, e.g. HEK293 cells. With this new system we are able to deliver up to about 2×1011 viral genomes per milliliter (vg/mL) of unconcentrated AAV vectors.

Abstract: The present invention harnesses the power of mutagenesis to produce an attenuated RNA virus in a very short period, i.e. as soon as the complete sequence of the target virus is known and an infectious genome can be produced.

Abstract: Provided are cytochrome P450 polypeptides, including cytochrome P450 santalene oxidase polypeptides, cytochrome P450 bergamotene oxidase polypeptides and cytochrome P450 reductase polypeptides. Also provided are nucleic acid molecules encoding the cytochrome P450 polypeptides. Cells containing the nucleic acids and/or the polypeptides are provided as are methods for producing terpenes, such as santalols and bergamotols, by culturing the cells.

Abstract: Provided herein are compositions and methods for the assembly of a tripartite or multipartite bioluminescent complex. In particular, a bioluminescent complex is formed upon the interaction of two or more peptide tags (e.g., separately or fused as a dipeptide or tripeptide) and a polypeptide component.

Abstract: Disclosed are a recombinant microorganism capable of producing methyl anthranilate, wherein the recombinant microorganism is obtained by introducing an aamt1 gene into a microorganism having the capacity to produce anthranilic acid (ANT), and a method of producing methyl anthranilate using the same. The recombinant microorganism is capable of producing methyl anthranilate using only a purely biological process from renewable carbon-circulating biomass without chemical catalytic reaction, thus having an advantage of enabling mass production of high value-added methyl anthranilate in a very economical manner based on an environmentally friendly and simple production process.

Abstract: Compositions and methods for conferring herbicide resistance or tolerance upon plants towards certain classes of herbicide are provided. In particular these are amine, alcohol and aminal herbicides. The compositions include nucleotide and amino acid sequences for wild-type and mutant glucosyl transferase polypeptides. The polypeptides of the invention are mutant or wild type glucosyl transferases that are capable of catalyzing the transfer of glucose to certain herbicidal structures and that, thereby, confer resistance or tolerance in plants to amine, alcohol and aminal PSII herbicides. Particularly, polypeptides of the invention include mutant or wild-type bx-type UDP glucosyl transferases.

Abstract: Disclosed are methods for isolating polymerase complexes from a mixture of polymerase complex components. The polymerase complexes can comprise a nanopore to provide isolated nanopore sequencing complexes. The methods relate to the positive and negative isolation of the polymerase complexes and/or nanopore sequencing complexes. Also disclosed is a nucleic acid adaptor for isolating active polymerase complexes, polymerase complexes comprising the nucleic acid adaptor, and methods for isolating active polymerase complexes using the nucleic acid adaptor.

Abstract: Provided are fusion proteins between p97 (melanotransferrin) and iduronate-2-sulfatase (IDS), and related compositions and methods of use thereof, for instance, to facilitate delivery of IDS across the blood-brain barrier (BBB) and/or improve its tissue penetration in CNS and/or peripheral tissues, and thereby treat and/or diagnose Hunter Syndrome (Mucopolysaccharidosis type II; MPS II) and related lysosomal storage disorders, including those having a central nervous system (CNS) component.

Abstract: The present invention relates to novel esterase, more particularly to esterase variants having improved thermostability compared to the esterase of SEQ ID No 1 and the uses thereof for degrading polyester containing material, such as plastic products. The esterases of the invention are particularly suited to degrade polyethylene terephthalate, and material containing polyethylene terephthalate.

Abstract: A modified endoinulinase is provided, comprising modified wild-type T. purpuregenus endoinulinase, or a functional fragment thereof, in which an amino acid residue at each one of one or more positions corresponding to 128, 316, 344, 350 or 504 of wild-type T. purpuregenus endoinulinase is substituted, wherein: (i) a tyrosine residue corresponding to Y128 is substituted with H, K or R; a glutamate residue corresponding to E344 is substituted with K, H or R; and a threonine residue corresponding to T504 is substituted with M, S or Y; and optionally an alanine residue corresponding to A316 is substituted with T, S, C or M; (ii) a tyrosine residue corresponding to Y128 is substituted with H, K or R; a glutamate residue corresponding to E344 is substituted with K, H or R; a threonine residue corresponding to T504 is substituted with M, S or Y; and a glutamine residue corresponding to Q350 is substituted with L, G, A, V or I; or (iii) a tyrosine residue corresponding to Y128 is substituted with H, K or R.

Abstract: The present invention relates to variants of an alpha-amylase having improved stability to chelating agents relative to its parent enzyme, compositions comprising the variants, nucleic acids encoding the variants, methods of producing the variants, and methods for using the variants.

Abstract: The present invention relates to alpha-amylase variants. The present invention also relates to polynucleotides encoding the variants; nucleic acid constructs, vectors, and host cells comprising the polynucleotides; and methods of using the variants.

Abstract: The present invention relates to variants of a parent glucoamylase having altered properties (e.g., improved thermostability and/or specific activity). In particular, the present invention provides compositions comprising the variant glucoamylases, including starch hydrolyzing compositions, animal feed compositions and cleaning compositions. The invention also relates to DNA constructs encoding the variants and methods of producing the glucoamylase variants in host cells.

Abstract: The present invention relates to a factor VII composition having a substantially homogeneous isoelectric point and to a method for formulating such a composition. The present invention also relates to the therapeutic use of a factor VII composition having a substantially homogeneous isoelectric point.

Abstract: The present invention provides for a polyketide synthase (PKS) capable of synthesizing an even-chain or odd-chain diacid or lactam or diamine. The present invention also provides for a host cell comprising the PKS and when cultured produces the even-chain diacid, odd-chain diacid, or KAPA. The present invention also provides for a host cell comprising the PKS capable of synthesizing a pimelic acid or KAPA, and when cultured produces biotin.

Abstract: Provided are a liquid enzyme preparation of transglutaminase and a preparation method thereof, wherein the liquid enzyme preparation is a liquid preparation of transglutaminase EC2.3.2.13, and its components and amounts thereof are as follows: the liquid preparation of transglutaminase has an enzyme activity of 10-1000 u/ml, a water activity regulator is present in an amount of 30-80 w/v %, a redox potential regulator is present in an amount of 0.0075-1 w/v %, a food preservative is present in an amount of 0-0.1 w/v %, and a pH regulator is added to a final volume of 100%. The preparation method thereof comprises purification of an enzyme solution, mixing, sterilization, filling, and obtaining of a final product.

Abstract: The present disclosure describes a nanoparticle comprising a three dimensional DNA nanocage and a payload biological macromolecule, and methods of assembly thereof.

Abstract: The invention provides barcode libraries and methods of making and using them including obtaining a plurality of nucleic acid constructs in which each construct comprises a unique N-mer and a functional N-mer and segregating the constructs into a fluid compartments such that each compartment contains one or more copies of a unique construct. The invention further provides methods for digital PCR and for use of barcode libraries in digital PCR.

Abstract: In an illustrative embodiment, automated multi-module cell editing instruments are provided to automate multiple edits into nucleic acid sequences inside one or more cells.

Abstract: The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA, Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene function. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response.

Abstract: The present invention relates to a polynucleotide comprising (i) a sequence encoding an anti-CRISPR (Acr) polypeptide and (ii) an miRNA target sequence and to vectors and host cells related thereto.

Abstract: A method of modifying a gene encoding or processed into a non-coding RNA molecule having no RNA silencing activity in a eukaryotic cell, with the proviso that said eukaryotic cell is not a plant cell, is disclosed. The method comprising introducing into the eukaryotic cell a DNA editing agent conferring a silencing specificity of said non-coding RNA molecule towards a target RNA of interest. A method of modifying a gene encoding or processed into a RNA silencing molecule to a target RNA in a eukaryotic cell is also disclosed. Methods of disease prevention and treatment, methods of inducing cell apoptosis and methods of generating a eukaryotic non-human organism are also disclosed.

Abstract: Therapeutic oligonucleotides comprising pharmacokinetic (PK)-modifying anchors are provided. Methods for treating diseases or disorders comprising administering to a subject a therapeutic oligonucleotide comprising one or more PK-modifying anchors are provided.

Abstract: Provided herein is a potent, optimized ?-catenin nucleic acid inhibitor molecule with a unique pattern of modified nucleotides. Also provided are methods and compositions for reducing ?-catenin expression and methods and compositions for treating cancer.

Abstract: Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for TTR.

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.

Abstract: The present disclosure pertains generally to chemically-modified oligonucleotides for use in research, diagnostics, and/or therapeutics. In certain embodiments, the present disclosure describes compounds and methods for the modulation of a target nucleic acid. In certain embodiments, the present disclosure describes compounds and methods for the modulation of Apoliprotein C-III expression.

Abstract: Methods and compositions are provided for oligonucleotide probes and oligonucleotide probe libraries that recognize targets of interest. The targets include circulating biomarkers such as microvesicles, including those derived from various diseases.

Abstract: Modified oligonucleotides comprising a GalNAc moiety of the present disclosure along with methods of making and use, e.g., against HBV are disclosed.

Abstract: A cancer-specific trans-splicing ribozyme and a use thereof are disclosed. The trans-splicing ribozyme does not act on normal tissue, but is specifically expressed in cancer tissue. Therefore, it is very safe and has excellent expression efficiency at the post-transcription level, and thus can be effectively used in treatment of cancer.

Abstract: Provided are delivery immunostimulatory bacteria that have enhanced colonization of tumors, the tumor microenvironment and/or tumor-resident immune cells, and enhanced anti-tumor activity. The immunostimulatory bacteria are modified by deletion of genes encoding the flagella or by modification of the genes so that functional flagella are not produced, and/or are modified by deletion of pagP or modification of pagP to produce inactive PagP product. As a result, the immunostimulatory bacteria are flagellin? and/or pagP?. The immunostimulatory bacteria optionally have additional genomic modifications so that the bacteria are adenosine and/or purine auxotrophs. The bacteria optionally are one or more of asst, purI? and msbB?.

Abstract: The present disclosure provides compositions and methods for treating, preventing, or inhibiting diseases of the eye. In one aspect, the disclosure provides HTRA1 RNAi agents and methods of using the same.

Abstract: The invention provides siRNA compositions that specifically downregulates expression of a variant of the PNPLA3 gene and methods of use thereof for treating a chronic liver disease or alcoholic liver disease (ALD).

Abstract: Described herein are devices and methods for increasing the production of steviol glycosides, which have industrial and economic value. The steviol glycosides produced by the devices and methods disclosed herein do not require the ultra purification that is common in conventional or commercial methods and do not have a bitter aftertaste, making them better suited as flavor-enhancing N additives to food, pharmaceutical, and nutritional supplement products.

Abstract: Methods for editing, e.g., introducing a heterologous transgene, within the human albumin gene (e.g., at intron 1) are provided.

Abstract: Compositions and methods for expressing alpha 1 antitrypsin (AAT) in a host cell are provided. Also provided are compositions and methods for treating subjects having alpha 1 antitrypsin deficiency (AATD).

Abstract: The invention relates to a method for producing a protein in a filamentous fungus cell, comprising the overexpression of the TrAZF1 gene or of one of the variants thereof in said cell.

Abstract: Epigenomic states of genome DNA are altered at multiple sites to rapidly change traits by providing a fusion protein including a first region that defines a polypeptide capable of binding sequence-specifically to multiple sites on genome DNA and a second region that defines a polypeptide capable of regulating an epigenomic state.

Abstract: The invention relates to the field of genetic engineering tools for gene expression in plants. Specifically, the invention concerns modified Foxtail Mosaic Virus (FoMV) vectors comprising polynucleotide sequences which are capable of driving expression of a gene of interest in a plant host. Accordingly, the invention concerns FoMV-based expression vectors comprising said polynucleotides, compositions comprising modified FoMV vectors, methods of generating gene expression in plants infected with the modified FoMV vectors. The expression vectors, compositions, plants and methods of the present invention find application in many fields of biotechnology, including, for example, gene characterization, protein production and agricultural biotechnology.

Abstract: Compositions and methods for regulating expression of heterologous nucleotide sequences in a plant are provided. Compositions include tissue-preferred promoters operably linked to morphogenic genes. Also provided is a method for expressing a heterologous nucleotide sequence in a plant using a promoter sequence disclosed herein operably linked to a morphogenic gene. DNA constructs comprising a promoter operably linked to a morphogenic gene and further comprising a heterologous nucleotide sequence of interest are also provided.

Abstract: Compositions and methods for providing desired cannabinoid content in cannabis plants. More particularly, the invention relates to compositions and methods for using cannabinoid synthase paralogs as guidance for breeding cannabis plants with a desired cannabinoid content, including but not limited to cultivars, varieties, lines and methods of breeding the same for commercial use.

Abstract: The present invention relates to a recombinant vector carrying cellulose-binding domain 3 and a method for separating a target protein using the recombinant vector. The protein isolating method of the present invention can easily separate a fusion protein containing a target protein by using the recombinant vector to bind a transformed plant body to cellulose and can effectively isolate the target protein from a cellulose-binding domain by treating the fusion protein with enterokinase, thus being expected to find industrial applications in various fields.

Abstract: The present invention refers to a plant or plant part comprising a polynucleotide encoding a mutated PPO polypeptide, the expression of said polynucleotide confers to the plant or plant part tolerance to herbicides.

Abstract: The invention pertains to the targeted alteration of a duplex DNA in a cell, whereby two site-specific nucleases generate an indel, such that the open reading frame is not altered after the second indel. The invention further pertains to the use of such nucleases for the targeted alteration of an open reading frame in duplex DNA and a kit of parts for use in a method of the invention. Using the method of the invention, novel plants were obtained having an improved herbicide resistance. The invention therefore also concerns plants having improved herbicide resistance due to the expression of an altered ALS protein.

Abstract: The present invention relates to a transgenic plant which integrated into its genome an exogenous polynucleotide encoding a polypeptide which confers resistance to one or more races of Puccinia graminis f. sp. tritici, such as the Ug99 group of races Puccinia graminis f. sp. tritici.

Abstract: A transgenic soybean plant resistant to soybean cyst nematode (SCN), or parts thereof, are provided. Also provided are methods of increasing SCN resistance of a soybean plant and associated DNA constructs.

Abstract: The invention provides a transgenic Gossypium hirsutum event MON 88702, plants, plant cells, seeds, plant parts, progeny plants, and commodity products comprising event MON 88702. The invention also provides polynucleotides specific for event MON 88702, plants, plant cells, seeds, plant parts, progeny plants, and commodity products comprising polynucleotides for event MON 88702. The invention also provides methods related to event MON 88702.

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, as probes for the isolation of other homologous (or partially homologous) genes. The insecticidal 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: The present invention provides a method for modifying a targeted site of a double-stranded DNA in a cell, the method including a step of bringing a complex in which a nucleic acid sequence-recognizing module that specifically binds to a selected target nucleotide sequence in a double-stranded DNA and a nucleic acid base converting enzyme or DNA glycosylase are linked, and a donor DNA containing an insertion sequence into contact with said double-stranded DNA, to substitute the targeted site with the insertion sequence, or insert the insertion sequence into said targeted site, without cleaving at least one strand of said double-stranded DNA in the targeted site.