Abstract: Disclosed are materials and methods for treating diseases of the mammalian eye, and in particular, Usher syndrome 1B (USH1B). The invention provides AAV-based, dual-vector systems that facilitate the expression of full-length proteins whose coding sequences exceed that of the polynucleotide packaging capacity of an individual AAV vector. In one embodiment, vector systems are provided that include i) a first AAV vector polynucleotide that includes an inverted terminal repeat at each end of the polynucleotide and a suitable promoter followed by a partial coding sequence that encodes an N-terminal portion of a full-length polypeptide; and ii) a second AAV vector polynucleotide that includes an inverted terminal repeat at each end of the polynucleotide and a partial coding sequence that encodes a C-terminal portion of a full-length polypeptide, optionally followed by a polyadenylation (pA) signal sequence.

Abstract: The present invention refers to a method for controlling undesired vegetation at a plant cultivation site, the method comprising the steps of providing, at said site, a plant that comprises at least one nucleic acid comprising a nucleotide sequence encoding a wild-type or a mutated protoporphyrinogen oxidase (PPO) which is resistant or tolerant to a PPO-inhibiting herbicide by applying to said site an effective amount of said herbicide. The invention further refers to plants comprising wild-type or mutated PPO enzymes, and methods of obtaining such plants.

Abstract: Provided herein are methods of delivering “split” Cas9 protein or nucleobase editors into a cell, e.g., via a recombinant adeno-associated virus (rAAV), to form a complete and functional Cas9 protein or nucleobase editor. The Cas9 protein or the nucleobase editor is split into two sections, each fused with one part of an intein system (e.g., intein-N and intein-C encoded by dnaEn and dnaEc, respectively). Upon co-expression, the two sections of the Cas9 protein or nucleobase editor are ligated together via intein-mediated protein splicing. Recombinant AAV vectors and particles for the delivery of the split Cas9 protein or nucleobase editor, and methods of using such AAV vectors and particles are also provided.

Abstract: The present invention provides recombinant adenoviral vectors, immunogenic compositions thereof and their uses in medicine. In particular, the present invention provides an adenoviral vector comprising the genome of an adenovirus other than AdHu5 and AdY25, wherein the genome of the adenovirus has been modified such that the vector lacks the native E4 locus of the adenovirus and comprises heterologous E4Orf1, E4Orf2 and E4Orf3 coding regions from AdY25.

Abstract: Two-component vaccine formulations and methods are contemplated where the vaccine has an adjuvant component and a therapeutic component. The therapeutic component comprises preferably a recombinant therapeutic virus encoding a therapeutic antigen while the adjuvant component comprises a non-host cell or immune stimulating portion thereof. Notably, use of the adjuvant component will result in significant uptake of the therapeutic component into immune competent cells, even in the absence of receptors for entry of the therapeutic component. In addition, such adjuvant also stimulates expression of the therapeutic antigen.

Abstract: The activity of a number of therapeutic antibodies is mediated in part by antibody-dependent cell-mediated cytotoxicity (ADCC). An engineered effector cell line expressing the low affinity Fc receptor, Fc?R111a (CD16), that responds to ligation of the Fc moiety of antibody bound to the specific antigen expressed on target cells by activation of a NFAT responsive reporter gene is described. In this cell line the firefly luciferase (FL) reporter gene is regulated by a novel synthetic chimeric promoter containing binding sites for NF-AT, AP1, NFkB, and STAT5 that confers improved sensitivity, an improved dynamic range, an improved tolerance to human serum and a reduced incubation time, relative to engineered effector cell lines that express a NFAT regulated reporter-gene, when used in an ADCC assay together with engineered target cells.

Abstract: Isolated cDNA sequences encoding for highly conserved domains of plant viral pathogen genomes. The cDNA sequences are selected from the group consisting of those listed in Table I (SEQ ID NOs: 1-68). cDNA fusion constructs or chimeric transgene constructs comprising at least two different cDNA sequences selected from the list in Table I, and more specifically cDNA corresponding to at least two different viruses is described, along with methods of creating transgenic plants with broad-spectrum, durable resistance to multiple viral pathogens using these cDNA sequences.

Abstract: The present invention provides a process for producing a food ingredient or beverage ingredient. The present invention also provides a process for producing food product or beverage product. The present invention also provides a process for providing starch, amylose, amylopectin, ?-glucan, fructan, non-starch polysaccharide, dietary fibre or resistant starch to improve one or more indicators of health in a mammal.

Abstract: This disclosure provides transgenic plants having enhanced traits such as increased yield, enhanced nitrogen use efficiency and enhanced drought tolerance; propagules, progeny and field crops of such transgenic plants; and methods of making and using such transgenic plants. This disclosure also provides methods of producing hybrid seed from such transgenic plants, growing such seed and selecting progeny plants with enhanced traits. Also disclosed are transgenic plants with altered phenotypes which are useful for screening and selecting transgenic events for the desired enhanced trait.

Abstract: The present invention provides plant virus vectors developed from the Sugarcane mosaic virus (SCMV). The vectors include a nucleic acid sequence encoding an infectious Sugarcane mosaic virus (SCMV) operably linked to one or more regulatory elements functional in a plant. The plant virus vectors may be used to infect monocot plants, such as maize, for gene expression applications.

Abstract: The present invention relates to a nanocarrier peptide sequence (SEQ ID NO: 6 KXPXXXXA/V/GXGNXX; wherein X is selected from amino acid R, K, A or H. The present invention also relates to the method for cellular delivery, by implementing the steps of: complexation of a peptide nanocarrier sequence: KXPXXXXA/V/GXGNXX; where X is selected from amino acid R,K,A and H having SEQ ID NO: 6 with a macromolecule to obtain a complex; and administering the complex to a targeted mammalian or plant cell or tissue.

Abstract: The present invention provides a method of detecting one or more Klebsiella species within a sample from a subject, the method comprising: subjecting DNA and/or RNA from the sample to a PCR amplification reaction using primer pairs targeting species-specific canonical single nucleotide polymorphisms (canSNPs); and analyzing amplification products resulting from the PCR amplification reaction to detect the one or more Klebsiella species. The present invention also provides a kit for detection of one or more Klebsiella species, Klebsiella clonal groups, AMR genes, and/or virulence genes, the kit comprising primer pairs targeting species-specific canSNPs, K. pneumoniae genes M1 and M2, clonal group-specific canSNPs, AMR genes, and/or virulence genes.

Abstract: The present disclosure provides, in some embodiments, compositions, kits, systems, and methods for reducing bacteria on a surface (e.g., a medical device) and preventing and/or treating a bacterial infection (e.g., urinary tract infection) in a subject using IdsD protein or a fragment thereof.

Abstract: Provided are a human PD-1 knockdown siRNA, a recombinant expression CAR-T vector, a preparation method thereof, and an application of the same. A PD-1 knockdown siRNA expression cassette and an siRNA expression product thereof can be applied to a CAR-T therapy of multiple myeloma (MM) for eliminating or alleviating a tumor immune escape mechanism, and in the suppression of an immune escape mechanism in a CAR-T therapy of a tumor, such as pancreatic cancer, brain glioma, and myeloma.

Abstract: A heterologous expression cassette, DNA construct and vaccine composition for immunization against flavivirus and/or other pathogens. DNA constructs, recombinant viruses and vaccine compositions containing the recombinant viruses were obtained. This invention also concerns and provide an improved expression vector of the live-attenuated yellow fever 17D virus. Modifications in the expression cassette of heterologous proteins in the intergenic E/NS1 region of the yellow fever 17D vaccine virus, were made. The two new functional domains inserted in the expression cassette were (1) a coding sequence for the N-glycosylation motif, located between the NS1 N-terminal motif and the heterologous protein and (2) a sequence which promoted the proteolytic cleavage, or not, of the recombinant protein in such a way as to release it from its C-terminal containing the transmembrane domains and, consequently, from its association with the membrane of the endoplasmatic reticulum—ER.

Abstract: Provided are genetically modified NK cells expressing a chimeric antigen receptor targeting an EGFR superfamily receptor. The CAR can comprise an intracellular domain of Fc?RI? and further recombinant proteins expressed by the genetically modified NK cells are CD16, autocrine growth stimulating cytokines, and optionally one of IL-12, a TGF-beta trap, or a homing receptor. Also described are methods for treating a patient having or suspected of having a disease that is treatable with NK-92 cells, such as cancer, comprising administering to the patient the genetically modified NK cells.

Abstract: The present invention relates to methods for controlling hybridization in plants and producing hybrid plants. The present invention also relates to nucleic acids encoding amino acid sequences for self-incompatibility (SI) proteins in plants, and the use thereof for the manipulation of SI, including seed production, in plants, particularly of the Poaceae family. The present invention also relates to kits, compositions, constructs and vectors including such nucleic acids, and related polypeptides, regulatory elements and methods. The present invention also relates to expression of self-gamete recognition genes in plants and to related nucleic acids, constructs, molecular markers and methods.

Abstract: Provided are nucleic acid constructs and systems which comprise (i) a first nucleic acid construct encoding a toxin operatively linked to a first promoter and at least one cancer-associated signaling responsive enhancer element; and (ii) a second nucleic acid construct encoding an anti-toxin operatively linked to a second promoter, the second promoter being stronger than the first promoter. Also provided are pharmaceutical compositions comprising same and methods of using same for treating cancer.

Abstract: Compositions and methods for improving plastid transformation in difficult to transform plants are disclosed.

Abstract: Described are methods and compositions for enhancing drought tolerance in plants. Nucleic acid constructs therefore are also described. Transgenic plants are also provided that exhibit enhanced agronomic properties. The inventors have demonstrated increased drought tolerance in connection with increased expression of the Xa21 gene.

Abstract: Antibodies which specifically bind to the Axl protein are described. Also disclosed are methods for the production and use of the anti-Axl antibodies.

Abstract: A method of treating a human patient comprising systemically administering multiple doses of a parenteral formulation of a replication capable oncolytic adenovirus of subgroup B in a single treatment cycle, wherein the total dose given in each dose is in the range of 1×1010 to 1×1014 viral particles, and wherein each dose of virus is administered over a period of 1 to 90 minutes, for example at a rate of viral particle delivery in the range of 2×1010 particles per minute to 2×1012 particles per minute. Also provided are formulations of the oncolytic adenoviruses and combination therapies of the viruses and formulations with other therapeutic agents.

Abstract: The present disclosure provides (a) vectors comprising a multi-antigen construct encoding two, three, or more immunogenic PAA polypeptides; (b) compositions comprising the vectors, (c) methods relating to uses of the vectors and compositions for eliciting an immune response or for treating prostate cancers.

Abstract: Some embodiments described herein relate to cells which have been genetically engineered to release a polypeptide when a population of the cells reaches a desired density. In some embodiments, the released polypeptide may be a therapeutic polypeptide. In some embodiments, the therapeutic polypeptide kills tumor cells or which inhibits the growth of tumor cells.

Abstract: The present invention relates to novel plants, in particular to pepper plants capable of producing fruits which can be kept a longer period of time on the plant as well as being stored after harvesting under refrigerated conditions without exhibiting excessive softening. The invention thus refers to pepper plant which produces fruits with significantly increased fruit firmness at the harvesting stage, wherein said increased fruit firmness is controlled by a genetic determinant, monogenic, wherein said increased fruit firmness is defined as a fruit deformation under a 1 kg load force that is lower than that of a fruit from a control pepper plant which does not have the said genetic determinant.

Abstract: HSV glycoprotein B-VSV-G1 fusion proteins and variants thereof, HSV glycoprotein C antigenic peptide constructs and variants thereof, HSV immunogenic compositions and protein vaccines including the peptide constructs, and HSV DNA vaccines encoding the amino acid sequence of the peptide constructs, as well as methods of using the vaccines and compositions comprising the vaccines.

Abstract: The present invention encompasses compositions and methods to allow one to monitor formation of synthetic chromosomes in real-time via standardized fluorescent technology, eliminating the need for cumbersome, expensive, and possibly mutagenic analysis.

Abstract: The present invention relates to the production of structural proteins and virus-like particles (VLPs) of flaviviruses and hepatitis C. Production is through use of a single expression cassette comprising a viral structural gene, a furin encoding gene and a bicistronic expression element such as an internal ribosome entry site (IRES) between the viral and furin genes. Both the viral gene and furin gene can include a partial capsid encoding sequence acting as a signal peptide to co-locate the viral protein and furin and to act as a membrane anchor for the viral protein and furin. A separate expression cassette comprising a non-structural viral gene can be combined with the initial cassette. The structural proteins and VLPs can be used in vaccines and in the treatment of viral infections.

Abstract: The present invention provides compositions and methods useful for treating disorders amenable to therapy via introduction of multigenic expression vectors. More particularly, the invention provides vectors and polynucleotides encoding polypeptides for treatment of cardiac disorders wherein said polypeptides may comprise a cytokine, a chemokine, and/or an angiogenic polypeptide, or functional derivatives thereof. Also provided, as compositions of the invention, are linkers useful for connecting and expressing functional (biologically active) polypeptides from single, multigenic-expression constructs.

Abstract: A method of producing a virus like particle (VLP) in a plant is provided. The method comprises introducing a first nucleic acid and a second nucleic acid into the plant, or portion of the plant. The first nucleic acid comprises a first regulatory region active in the plant and operatively linked to a nucleotide sequence encoding a structural virus protein. The second nucleic acid comprises a second regulatory region active in the plant and operatively linked to a nucleotide sequence encoding a channel protein, for example but not limited to a proton channel protein. The plant or portion of the plant is incubated under conditions that permit the expression of the nucleic acids, thereby producing the VLP.

Abstract: The present invention relates to a cell comprising (aa) a nucleic acid comprising in 5? to 3? direction (i) at least one adeno-associated virus (AAV) inverted terminal repeat (ITR) sequence; (ii) a promoter which is capable of being activated by (a) helper polypeptide(s) and optionally (a) helper polynucleotide(s); and (iii) a transgenic coding sequence under the control of said promoter of (aa)(ii); and (ab) a nucleic acid comprising in 5? to 3? direction (i) a promoter which is capable of being activated by said helper polypeptide(s) and optionally said helper polynucleotide(s); and (ii) at least one AAV rep gene coding sequence under the control of said promoter of (ab)(i); wherein said cell does not comprise an AAV cap gene and/or is not able to express any AAV cap gene product.

Abstract: Provided herein are a glucan kinase polypeptide, an isolated polynucleotide, and a method for processing starch. The glucan kinase polypeptide comprises an isolated polypeptide including a sequence selected from the group consisting of SEQ ID NO: 2, SEQ ID NO:22, fragments thereof, variants thereof, and combinations thereof. The isolated polynucleotide comprises a nucleotide sequence selected from the group consisting of SEQ ID NO: 1, SEQ ID NO: 21, a fragment thereof, a variant thereof, and a combination thereof. The method for processing starch comprises providing a glucan dikinase; exposing a starch to the glucan dikinase; and collecting the starch that has been exposed to the glucan dikinase.

Abstract: Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification 5 conferring stability to the RNA, as well as related methods, are disclosed.

Abstract: The present invention relates to a method of obtaining a transformed plant cell. The present invention comprises the steps of: (a) co-transforming an intended DNA and a first marker gene into a plant cell; and (b) selecting from the transformed cells obtained in the step (a), a transformed plant cell wherein the intended DNA is introduced into a chromosome thereof, and the first marker gene is not introduced, wherein the method does not contain a step to exclude a transformed cell with only the intended DNA introduced into the chromosome by positive selection using the first marker gene.

Abstract: Compositions for reducing fucosylation of glycoproteins in insect cells are provided. Also disclosed are methods of use of such compositions for the production of recombinant humanized proteins.

Abstract: The present disclosure provides methods and compositions useful for the treatment or prevention of heart disease. In particular, the present disclosure provides a vector comprising a modified troponin T promoter operatively linked to a therapeutic gene product for the treatment or prevention of heart disease, e.g., cardiomyopathy. The gene product may be MYBPC3. The disclosure also provides recombinant adeno-associated virus (rAAV) virions, rAAV viral genomes, and expression cassettes and pharmaceutical compositions thereof. The disclosure further provides methods for treating a disease or disorder, such as heart disease.

Abstract: A method of treating a neurological disease (such as autism) in a subject is disclosed. The method comprises administering to the subject a therapeutically effective amount of microparticles derived from mesenchymal stem cells.

Abstract: A baculovirus displaying a porcine epidemic diarrhea virus S protein or S1 domain thereof is provided for preventing porcine epidemic diarrhea virus infection.

Abstract: The present invention relates to a retroviral system for the transfer of non-viral RNA into target cells and more particularly a retroviral particle capable of delivering multiple RNAs. More particularly, it relates to retroviral particles comprising a protein derived from the Gag polyprotein an envelope protein, optionally an integrase and at least two encapsidated non-viral RNAs, the encapsidated non-viral RNAs each comprising an RNA sequence of interest linked to an encapsidation sequence, each encapsidation sequence being recognised by a binding domain introduced into the protein derived from the Gag polyprotein and/or into the integrase.

Abstract: Embodiments of the invention provide a method of detecting one or more strains of Klebsiella pneumoniae. The method may include forming a plurality of mixtures for nucleic amplification. The method can include amplification of specific sequences within the K. pneumonia genome that can provide definitive information to distinguish between one or more types or strains of K. pneumonia.

Abstract: The present invention relates to nucleic acid molecules containing spacers that can be packaged into viral particles and methods of producing them. In a first aspect, the invention features a nucleic acid molecule including a first spacer (SSI); a first inverted terminal repeat (ITR1); a cloning site (CS); a second inverted terminal repeat (ITR2); and a second spacer (SS2), such as a eukaryotic spacer; operably linked to each other in a 5?-to-3? direction as: SS1-ITR1-CS-ITR2-SS2. In an embodiment, the invention features a vector comprising any of the above-described nucleic acid molecules. In another aspect, the invention features a plurality of viral particles including the nucleic acid molecule. The invention further includes a host cell including any of the above-described vectors.

Abstract: Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes a survival factor, wherein the genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or a component or a transcriptional regulator of a MHC-I or MHC-II complex, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor.

Abstract: Compositions and methods for regulating expression of heterologous nucleotide sequences in a plant are provided. Compositions include nucleotide sequences for regulatory regions. Phospholipid transfer protein (PLTP) promoters from maize are provided. Also provided is a method for expressing a heterologous nucleotide sequence in a plant using a promoter sequence, such as a maize PLTP promoter, disclosed herein. DNA constructs comprising a promoter operably linked to a heterologous nucleotide sequence of interest are also provided.

Abstract: The present invention relates to antibodies or fragments thereof that specifically bind Fc?RIIB, particularly human Fc?RIIB, with greater affinity than the antibodies or fragments thereof bind Fc?RIIA, particularly human Fc?RIIA. The present invention also provides the use of an anti-Fc?RIIB antibody or an antigen-binding fragment thereof, as a single agent therapy for the treatment, prevention, management, or amelioration of a cancer, preferably a B-cell malignancy, particularly, B-cell chronic lymphocytic leukemia or non-Hodgkin's lymphoma, an autoimmune disorder, an inflammatory disorder, an IgE-mediated allergic disorder, or one or more symptoms thereof. The invention provides methods of enhancing the therapeutic effect of therapeutic antibodies by administering the antibodies of the invention to enhance the effector function of the therapeutic antibodies. The invention also provides methods of enhancing efficacy of a vaccine composition by administering the antibodies of the invention.

Abstract: Provided herein are polypeptides comprising one or more non-native cysteine residues that form a disulfide bridge between non-native cysteines within the protein or between non-native cysteines of two monomers of the protein. Such modified human polypeptides are useful in treatment of genetic diseases via enzyme replacement therapy and/or gene therapy.

Abstract: Methods and compositions for modifying the coding sequence of endogenous genes using rare-cutting endonucleases and transposases. The methods and compositions described herein can be used to modify the coding sequence of endogenous genes.

Abstract: Disclosed herein are transfection complexes comprising at least one cell surface ligand; at least one helper lipid component; and a transfection enhancer. Also disclosed are pharmaceutical compositions comprising the disclosed transfection complexes, and a pharmaceutically acceptable carrier. Further, disclosed are methods of transfecting a cell, the method comprising the steps of: obtaining a transfection complex as disclosed; and contacting a cell with the transfection complex.

Abstract: The present invention is directed to an in vitro method of producing a recombinant AAV virion in a mammalian host cell that comprises a functional adenoviral E1A gene and rAAV virions made by the method. Host cells are transfected with varying ratios of plasmids comprising the E1A gene and virions.

Abstract: The invention provides CadA polypeptides with mutations that increase activity in alkaline pH compared to the wild-type lysine decarboxylase. The invention also provides methods of generating such mutant polypeptides, microorganisms genetically modified to overexpress the mutant polypeptides, and methods of generating such microorganisms.

Abstract: The present disclosure relates to the genetically modified non-human animals that express a human or chimeric CTLA-4, and methods of use thereof.