Abstract: Provided are transgenic mice whose genome comprises a nucleic acid sequence encoding human cereblon (CRBN) or a fragment thereof, wherein endogenous mouse CRBN is not expressed in the transgenic mice. Also provided are cells, cell lines, tissues, and organs derivable from the transgenic mice, and methods for producing and using such mice.

Abstract: A system and method for controlling metabolic enzymes or pathways in cells to produce a chemical above the levels of a wild-type strain is disclosed. The system utilizes cells, including yeasts, bacteria, and molds, having at least two genes capable of being controlled bi-directionally with light, where one gene is turned from off to on when exposed to light and another gene is turned from on to off when exposed to light, the two genes reversing when the light is turned off. Cells may utilize any number of sequences that benefit chemical production, including sequences that: encode for constitutive transcription of light-activated transcription factor fusions; encode for a metabolic enzyme; encode for a repressor; induce expression of metabolic enzymes; and an endogenous or exogenous activator expressed by a constitutive promoter, inducible promoter, or gene circuit.

Abstract: Disclosed herein is a vaccine comprising a Middle East Respiratory Syndrome coronavirus (MERS-CoV) antigen. The antigen can be a consensus antigen. The consensus antigen can be a consensus spike antigen. Also disclosed herein is a method of treating a subject in need thereof, by administering the vaccine to the subject.

Abstract: This disclosure relates to an animal model of human disease. More specifically, this disclosure relates to a rodent model of mood disorders such as unipolar depression and an anxiety disorder. Disclosed herein are genetically modified rodent animals that carry a humanized G protein-coupled receptor 156 (GPR156) gene that encodes a mutant human GPR156 protein comprising Asp at an amino acid position corresponding to position 533 in a full length wild type human GPR156 protein.

Abstract: Provided herein are systems, methods and compositions for rendering cells or the expression of an effector protein sensitive to a predetermined condition. In one aspect, cells can be rendered dependent upon the presence of an environmental agent, e.g., an exogenous agent, without which the cell will default to expression of a death protein and be killed. In another aspect, cells can be rendered sensitive to the presence of a set of predetermined conditions such that cells will only grow when two or more necessary exogenous agents are supplied, and without either of which, the cells are killed. In this aspect, hybrid transcription factors provide a vast array of possible predetermined conditions.

Abstract: The present invention relates to a tissue-specific promoter system for expressing microRNA (miRNA) for RNA interference-based methods of gene therapy. In these systems, the miRNA will inhibit gene expression or replace natural miRNA expression using microRNA.

Abstract: The present disclosure relates to genetically modified non-human animals that express a human or chimeric (e.g., humanized) IL15, and methods of use thereof.

Abstract: The present invention relates to polypeptides and more particularly to Transcription Activator-Like Effector derived proteins that allow to efficiently target and/or process nucleic acids. Particularly, the present invention reports the characterization of TALE derived proteins that can efficiently target methylated DNA. The present invention more specifically relates to TALE derived proteins that allow activation of methylated promoters responsible for gene silencing.

Abstract: This disclosure is directed to novel CD133-binding agents. The disclosure is also directed to uses of novel CD133-binding agents for detecting CD133-expressing cells and/or quantitating levels of cellular CD133 expression, for targeting CD133-expressing cells, for decreasing levels of CD133 in CD133-expressing cells and for treating or preventing cancer.

Abstract: Novel lentivirus packaging systems engineered with a synthetic gene network having a positive feedback loop to amplify the expression of virus genes are provided. When co-transfected into a host cell with a transfer plasmid and envelope vector, extremely high viral titers are achieved when compared to transfection of a host cell with conventional third generation packaging systems. Methods for enhancing production of lentivirus, compositions comprising high titer lentivirus, and therapeutic methods based on delivery of lentiviral nucleic acid to target cells are also provided.

Abstract: The present invention relates to new Transcription Activator-Like Effector proteins and more particularly new Transcription Activator-Like Effector Nucleases (TALENs) that can efficiently target and process nucleic acids. The present invention also concerns methods to use these new Transcription Activator-Like Effector proteins. The present invention also relates to vectors, compositions and kits in which Transcription Activator-Like Effector proteins of the present invention are used.

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.

Abstract: An aquacultured shrimp contains carotenoid and does not have a broken 2nd antenna.

Abstract: Disclosed herein, are compositions and methods for the treatment of human immunodeficiency virus infection. The compositions comprise engineered transcription activator like effector nucleases (TALENs) comprising a TALE DNA binding domain flanked by two spacer sequences, and a Fokl nuclease catalytic domain. Also, described herein, are methods of using TALENs to cleave nucleic acids; and methods of administering the TALENs to subjects at risk for or having an HIV infection.

Abstract: The invention is directed to transcription activator-like effector nuclease (TALEN)-mediated DNA editing of disease-causing mutations in the context of the human genome and human cells to treat patients with compromised genetic disorders.

Abstract: A method of introducing naked dsRNA into a seed is provided. The method comprising contacting the seed with the naked dsRNA under conditions which allow penetration of the dsRNA into the seed, thereby introducing the dsRNA into the seed.

Abstract: The present disclosure relates to the genetically modified non-human animals that express a human or chimeric (e.g., humanized) Lymphocyte Activation Gene 3 (LAG-3), and methods of use thereof.

Abstract: Proteins (20) having glucose-binding sites (28) that bind to glucose (30) are described.

Abstract: The present invention provides compounds, compositions thereof, and methods of using the same.

Abstract: The invention provides a process of isolating or enriching a heavy metal present in a liquid medium. The process comprising the following steps: (a) incubating a suspension containing (i) particulate scrap metal, household waste and/or industrial waste containing a heavy metal in elemental form and (ii) biomass comprising a bacterium, or a combination of two or more bacteria, capable of binding the heavy metal; (b) separating the biomass having bound heavy metal from the suspension of step (a); and (c) isolating the heavy metal from the biomass separated in step (b).

Abstract: The current invention provides a modular vector system that enables the insertion of gene expression cassettes in a recursive directional stacking fashion by rare restriction sites which requires only one type of vector. The invention also provides DNA molecules, compositions, and transgenic organisms, plants, plant tissues, plant seeds, and cells comprising recombined restriction sites for rare restriction enzymes.

Abstract: The present disclosure provides improved genome editing compositions and methods for editing a CBLB gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.

Abstract: The present invention is directed to a cellular biosensor system comprising (A) a repressor module comprising one or more genes, which in their cumulative gene action exert repressing and/or inhibitory effect(s) on (B), an output module comprising at least one gene comprising at least one output sequence generating one or more output signals (i) in the absence of repressing and/or inhibitory effect(s) of the repressor module (A) and (ii) in the presence of at least one recombinase expressed by (C), a recombinase module comprising at least one gene comprising at least one sequence encoding a site-specific recombinase that enables gene rearrangement in the output module resulting in one or more output signals in the absence of repressing and/or inhibitory effect(s) of the repressor module, wherein the repressing and/or inhibitory effects of the repressor module are controlled by one or more inputs that negatively affect the repressing and/or inhibitory effects of the repressor module (A).

Abstract: Provided herein are specific CD40 receptor agonist proteins, nucleic acids encoding the same, and methods of treating a subject having a CD40L-associated disease or disorder. The CD40 receptor agonist proteins provided herein comprise three soluble CD40L domains and an Fc fragment. The CD40 receptor agonist proteins are substantially non-aggregating and suitable for therapeutic, diagnostic and/or research applications.

Abstract: The present invention provides Factor IX (FIX) fusion proteins comprising at least one heterologous moiety, such as an XTEN. The present invention further discloses methods of making and using the FIX fusion proteins.

Abstract: A GMP adapted to provide the PAL gene for the treatment of PKU when administered orally. The GMP of the present invention may include a probiotic, a PAL gene to be expressed using the probiotic, wherein the PAL gene is functionally attached to a promoter and a ribosome binding site, and may be codon-optimized for expression in a certain host organism. A method of treating the metabolic disease of PKU by oral administration and ingestion of a GMP is also provided.

Abstract: A bivalent siRNA chimera platform capable of efficiently delivering and silencing two or more genes in vivo or in vitro is provided. Methods of using the bivalent siRNA chimeras for selectively targeting cells to down-regulate the expression of multiple genes are also provided.

Abstract: In accordance with the invention, isolated nucleic acids, expression methods, host cells, expression vectors, and DNA constructs for producing proteins, and proteins produced using the expression methods are described. More particularly, nucleic acids isolated from Pichia pastoris wherein the nucleic acids have promoter activity are described. The invention also relates to expression methods, host cells, expression vectors, and DNA constructs, for using the Pichia pastoris promoters to produce proteins and polypeptides, and to the proteins and polypeptides produced using the expression methods.

Abstract: Non-mammalian, transgenic animals, e.g., flies, that include a RAS transgene, are provided. Also provided are methods of using the subject transgenic non-mammalian animals to identify compounds having activity with respect to cellular proliferative, such as neoplastic, diseases.

Abstract: The present invention relates to the production of hydrolyzates from a lignocellulose-containing material, and to fermentation of the hydrolyzates. More specifically, the present invention relates to the detoxification of phenolic inhibitors and toxins formed during the processing of lignocellulose-containing material by enzymatically sulfating the phenolic inhibitors and toxins using aryl sulfotranseferases.

Abstract: The present disclosure generally relates to methods and compositions for contraception. In some embodiments, vector based approaches for contraception are provided.

Abstract: The invention relates to transgenic plants comprising an inverted-repeat construct which triggers post-transcriptional gene silencing of an endogenous visual reporter gene driven by a tissue-specific promoter wherein said tissue is relevant for pathogen entry, propagation or replication and their uses for screening natural or synthetic molecules, microorganisms or extracts from micro- or macro-organisms for their potential ability to inhibit pathogen entry, propagation or replication in plants by enhancing PTGS or for characterizing the mode of action of natural or synthetic molecules that are known to enhance plant disease resistance through an ill-defined mode of action.

Abstract: The present invention relates to a human artificial chromosome which is genetically transmissible to the next generation with high efficiency and the method for using the same. More specifically, the present invention relates to: a human artificial chromosome in which an about 3.5 Mb to about 1 Mb region containing an antibody ? light chain gene derived from human chromosome 22 is bound to a chromosome fragment which is transmissible to a progeny through a germ line of a non-human animal, said chromosome fragment is derived from another human chromosome; a non-human animal carrying the human artificial chromosome and an offspring thereof; a method for producing the non-human animal; a method for producing a human antibody using the nonhuman animal or an offspring thereof; and a human antibody-producing mouse carrying the human artificial chromosome.

Abstract: The present disclosure provides antibody drug conjugates that bind human cMET, their methods of making, and their uses to treat patients having cancer.

Abstract: Methods for improving or modulating targeting specificity of TALE proteins by introducing alternative RVDs into their modular nucleic acid binding domains. Polynucleotides encoding TALE proteins having alternative targeting specificity towards a nucleic acid target sequence. TALE proteins having alternative targeting specificity towards a nucleic acid target sequence and methods of making and using them.

Abstract: Genetically encoded, photocleavable proteins are derived from a fluorescent protein. Upon illumination, the proteins photocleave and spontaneously dissociate into two or more fragments or release one end of an internal loop.

Abstract: Pericytes are mural cells of brain capillaries that degenerate in multiple neurological disorders. Pericytes regulate neurovascular functions, but their role in the adult brain and disease is still poorly understood because of the lack of adequate pericyte-specific experimental models. All current pericyte-deficient models are not pericyte specific, and carry an inherited embryonic trait. Here, the Inventors generated an inducible pericyte-specific Cre line using a double-promoter strategy. The Inventors ablated adult mouse pericytes expressing Cre-dependent diphtheria toxin receptor after toxin administration. Pericyte ablation led to a rapid dysregulation of cerebral blood flow and blood-brain barrier breakdown. This was followed by behavioral deficits and neurodegenerative changes. These findings show that circulatory deficits leading to secondary neurodegeneration develop immediately after pericyte loss.

Abstract: The invention relates to a method for in vitro investigating mitochondrial replication dysfunction in a biological sample removed from a subject susceptible of suffering from physiological ageing or physiopathological conditions related to physiological ageing, or physiopathological ageing or associated symptoms or conditions, in particular premature ageing or accelerated ageing, or of a progeroid syndrome, such as Cockayne syndrome (CS), or neurodegenerative disorders or symptoms thereof, in which the levels of at least one species selected in the group of: POLG1 protein, POLG1 RNA, POLG2 protein, protease(s) which have POLG as a target, in particular serine protease(s) such as HTRA3 protein, HTRA2 protein and, HTRA3 RNA or HTRA2 RNA, or any combination of these species, are investigated.

Abstract: The present invention relates to polypeptides and more particularly to Transcription Activator-Like Effector derived proteins that allow to efficiently target and/or process nucleic acids. Particularly, the present invention reports the characterization of TALE derived proteins that can efficiently target methylated DNA. The present invention more specifically relates to TALE derived proteins that allow activation of methylated promoters responsible for gene silencing.

Abstract: The present disclosure describes the role for miR-322(424)/503 in the differentiation of cardiac precursor cells. Thus, the use of these molecules in the programming of resident stem/progenitor cells into cardiomyocytes, both in vitro and in vivo. Such methods find particular use in the treatment of patients post-myocardial infarction to prevent or limit scarring and to promote myocardial repair.

Abstract: A method of introducing naked dsRNA into a seed is provided. The method comprising contacting the seed with the naked dsRNA under conditions which allow penetration of the dsRNA into the seed, thereby introducing the dsRNA into the seed.

Abstract: The invention is directed to transcription activator-like effector nuclease (TALEN)-mediated DNA editing of disease-causing mutations in the context of the human genome and human cells to treat patients with compromised genetic disorders.

Abstract: The present invention provides an artificial nuclease comprising a DNA-binding domain and a function domain linked to each other via a polypeptide consisting of 35 to 55 amino acid residues wherein amino acid residues at two sites in a DNA-binding module contained in a DNA-binding domain exhibit a mode of repetition that is different for every four DNA-binding modules; a vector for expressing said artificial nuclease; a vector library for preparing said vector; and a vector set for preparing said vector library.

Abstract: A method is disclosed of predicting cancer patient response to immune checkpoint inhibitors, e.g., an antibody drug blocking ligand activation of programmed cell death 1 (PD-1) or CTLA4. The method includes obtaining mass spectrometry data from a blood-based sample of the patient, obtaining integrated intensity values in the mass spectrometry data of a multitude of pre-determined mass-spectral features; and operating on the mass spectral data with a programmed computer implementing a classifier. The classifier compares the integrated intensity values with feature values of a training set of class-labeled mass spectral data obtained from a multitude of melanoma patients with a classification algorithm and generates a class label for the sample. A class label “early” or the equivalent predicts the patient is likely to obtain relatively less benefit from the antibody drug and the class label “late” or the equivalent indicates the patient is likely to obtain relatively greater benefit from the antibody drug.

Abstract: The invention concerns a non-human animal model useful for sensitively studying gene-gene interactions over a wide genetic background; methods for producing the animal model; and methods for studying gene-gene interactions using an animal model of the invention.

Abstract: Luciferases which are different from those known heretofore have been desired. A luciferase mutant comprising an amino acid sequence in which at least one amino acid selected from the group consisting of valine at the position of 44, alanine at the position of 54 and tyrosine at the position of 138 is substituted with other amino acid(s) in the amino acid sequence of SEQ ID NO: 2.

Abstract: The invention relates to large-scale production of human antibodies by transgenic animals with high production of fully human IgG up to >10 g/L in sera with human IgG1 subclass dominancy. This invention also supports a feasibility of complex chromosome engineering for complicated genetic studies in non-murine mammalian species.

Abstract: Provided is a method for producing ambrein, comprising reacting tetraprenyl-?-curcumene cyclase with 3-deoxyachilleol A to obtain ambrein.

Abstract: The present invention relates to methods and compositions for high content drug screening in Caenorhabditis elegans which may be used to identify compounds that treat disorders associated with protein aggregation. It is based, at least in part, on the discovery that Caenorhabditis elegans, genetically modified to create a model system for disorders of protein aggregation, could be used, in a high throughput screening system, to identify agents that reduce the amount of aggregated protein.

Abstract: A dietary supplement includes comprising a therapeutic amount of astaxanthin derived from a synthetic or natural ester or diol, and at least one of a phospholipid, glycolipid, and sphingolipid, and formulated into an oral dosage form, wherein the astaxanthin is 0.1 to 15 percent by weight of the at least one phospholipid, glycolipid, and sphingolipid. The composition includes 0.5 to 12 mg of astaxanthin and may be used to treat low density lipoprotein (LDL) oxidation in humans and other diseases, disorders and impairments.