Abstract: Methods for derivatization of biomolecules including glycans or other biopolymers with one or more fluorescent, MS active compounds by reductive amination or rapid tagging in order to produce derivatized glycan having a pKa>7 and between about 200 ?2 and about 1000 ?2 of nonpolar surface area are described.

Abstract: The present invention relates to a library of particles, the library displaying a plurality of different T cell receptors (TCRs), wherein the plurality of TCRs consists essentially of TCRs comprising an alpha chain comprising an alpha chain variable domain and a beta chain comprising a beta chain variable domain and the library comprises more than one TRAV gene product and/or more than one TRBV gene product, wherein the beta chain variable domain does not comprise one or more of a TRBV5-1, 5-3, 5-4, 5-5, 5-6, 5-7 or 5-8 gene product and wherein the plurality of TCRs do not consist essentially of TCRs comprising a TRAV12-2 gene product from a natural repertoire and a TRBV6 gene product from a natural repertoire and TCRs comprising a TRAV21 gene product from a natural repertoire and a TRBV6 gene product from a natural repertoire.

Abstract: The invention relates to a library for the expression of all functional TCR types comprising 45 TCR constructs each encoding one of the 45 different TCR ? chains and 47 TCR constructs each encoding one of the 47 different TCR ? chains, wherein each of the 45 TCR constructs encoding one of 45 different TCR ? chain comprises the following building blocks one of the variable AV segments AVseg1 to AVseg45, and a constant AC segment, and wherein each of the 47 TCR constructs encoding one of 47 different TCR ? chains comprises one of the variable BV segments BVseg1 to BVseg47, and a constant BC segment.

Abstract: The present invention provides an in vitro blood vessel model for investigation of drug induced vascular injury and other vascular pathologies. The in vitro blood vessel model provides two channels separated by a porous membrane that is coated on one side by an endothelial cell layer and is coated on the other side by a smooth muscle cell layer, wherein said model is susceptible to the extravasation of red blood cells across said porous membrane due to drug induced vascular injury.

Abstract: The present disclosure provides high affinity and enhanced affinity T cell receptors specific for human Wilms tumor protein 1 (WT-1) epitopes for use in treating diseases or disorders, such as cancer cells that overexpress WT-1.

Abstract: The present invention concerns materials and methods for characterizing monoclonal immunoglobulin specificity of a Monoclonal Gammopathy of Undetermined Significance (MGUS) or Myeloma patients using a protein microarray comprising (a) a substrate, (b) antigens immobilized on the substrate, said antigens being selected from a defined group consisting of infectious agent antigens and/or self-antigens. In particular said protein microarray may be used to improve diagnosis, for the prognosis of myeloma or MGUS, for preventing transformation of MGUS toward myeloma, for adapting treatment of MGUS and myeloma or for monitoring the response to therapy of MGUS and myeloma patients.

Abstract: The present invention relates to mutated tissue plasminogen activators, and their use for treating thrombotic diseases.

Abstract: Provided herein are isolated populations of kidney cells harvested from differentiated cells of the kidney, wherein cells have been expanded in vitro, and methods of use thereof. The cells may be provided in a three dimensional matrix for culturing in vitro and/or implanting in vivo. Methods of seeding cells onto the matrix are also provided.

Abstract: A method for the analysis of samples including one or more glycopeptides including the steps of separating one or more glycopeptides using a chromatography system to produce a chromatographic eluent, adding a supercharging reagent to the chromatographic eluent, providing the chromatographic eluent and supercharging reagent to a mass spectrometer, ionizing the chromatographic eluent and supercharging reagent in an ion source to produce glycopeptide ions, performing at least one ion ion reaction on at least some of the glycopeptides ions to produce fragment ions, mass analyzing the fragment ions to produce ion ion reaction mass spectral data, and interpreting the ion ion reaction mass spectral data to provide structural information relating to the glycopeptide.

Abstract: Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.

Abstract: The invention concerns the field of cell culture technology. It concerns RNA having a specific sequence, expression vectors encoding said RNA, production host cell lines comprising said RNA, and methods of producing recombinant biopharmaceutical products using engineered host cell with altered levels of said RNAs, such as small non-coding RNAs, preferably microRNAs (miRNAs). The invention also relates to engineered host cells with altered levels in one or more of said RNAs. Those cell lines have improved secretion and/or growth characteristics in comparison to control cell lines.

Abstract: The invention provides for compositions and methods for identifying and validating modulators of cell fate, such as such as maintenance, cell specification, cell determination, induction of stem cell fate, cell differentiation, cell dedifferentiation, and cell trans-differentiation. The invention relates to reporter nucleic acid constructs, host cells comprising such constructs, and methods using such cells and constructs. The invention relates to methods for making cells comprising one or more reporter nucleic acid constructs using fluorogenic oligonucleotides. The methods relate to high throughput screens.

Abstract: Sequences of a serotype 8 adeno-associated virus and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles.

Abstract: Isolated monomeric aminoacyl-tRNA synthetase polypeptides and polynucleotides having non-canonical biological activities are provided, as well as compositions and methods related thereto.

Abstract: Provided herein are flu hemagglutinin polypeptides, including chimeric influenza virus hemagglutinin polypeptides, and flu hemagglutinin polypeptides comprising modified glycosylation sites and non-naturally glycosylation sites, compositions comprising the same, vaccines comprising the same and methods of their use.

Abstract: The present invention relates to a fusion protein comprising a skin-penetrating peptide, a polynucleotide encoding the fusion protein, an expression vector comprising the polynucleotide, a transformant comprising the expression vector, a method for preparing the fusion protein, a cosmetic composition for improving skin conditions, which comprises the fusion protein, and a pharmaceutical composition for external skin use, which comprises the fusion protein. The fusion protein of the invention comprises a skin-penetrating peptide bound to a physiologically active protein. The fusion protein significantly enhances the skin penetration and skin retention of the physiologically active protein while maintaining or enhancing the ability of the physiologically active protein to synthesize a material showing physiologically active effects. Thus, it can be widely used as an active ingredient in functional cosmetic compositions and pharmaceutical compositions for external skin use.

Abstract: Provided is a renal sarcomatoid cell line RCC09HYF, of which the deposit No. is CCTCC C201130, and the preparation method of the renal sarcomatoid cell line. The renal sarcomatoid cell line RCC09HYF can grow for a long period and be steadily passaged in vitro. By tumorigenic experiments using in-situ animal models in vitro it has been found that: the tumorigenesis is relatively fast inside animals and 3-4 weeks after tumor inoculation, the transplanted tumors fill the whole abdominal cavity, and dyscrasia appears in above 50% of nude mice; moreover, lung metastasis is present in a few individuals. The renal sarcomatoid cell line RCC09HYF can provide an effective and steady cell model for further study of the genesis and metastasis mechanism of renal sarcomatoid carcinoma in persons of Han nationality and for clinical prediction, diagnosis and treatment.

Abstract: Gene encoding human glucokinase mutant is provided. The gene has the nucleotide sequence chosen from the nucleotide sequence listed as SEQ ID NO:2 and the nucleotide sequence wherein the ORF region encodes the same amino acid sequence as the one encoded by ORF region (position 487 to 1884) of SEQ ID NO:2 and the rest of the region is same as the non-ORF region of SEQ ID NO:2. Human glucokinase mutant encoded by the gene, the recombinant vectors carrying the gene, the hosts comprising the vectors, pharmaceutical compositions thereof, uses thereof, and methods for treating and preventing diseases by using the same are provided. The human glucokinase mutant encoded by the gene has higher activity than that of the wild type human glucokinase, and thus provides a new way of controlling blood glucose and/or preventing and/or treating disturbance of carbohydrate metabolism, especially preventing and treating diabetes.

Abstract: The present invention refers to single-chain fusion proteins comprising three soluble TNF superfamily (TNFSF) cytokine domains and nucleic acid molecules encoding these fusion proteins. The fusion proteins are substantially non-aggregating and suitable for therapeutic, diagnostic and/or research applications.

Abstract: The invention pertains to a novel cell line, an HIV tat-rev dependent GFP-Gaussia luciferase Reporter cell line, known henceforth as the GGR cell line, that detects pseudotype and replication competent HIV (cloned or uncloned isolates, in cell media or human serum) rapidly and with high sensitivity. This GGR cell line provides an improved method of characterizing the entry phenotype of HIV envelope genes, and detecting and examining primary HIV samples in the context of laboratory research, clinical trial monitoring, and medical diagnostics. Examples include, but are not limited to, determining the functional HIV viral load, responsiveness to treatment, characterization of viral co-receptor usage (testing for viral co-receptor usage, i.e., CCR5 vs CXCR4, as required prior to prescribing FDA-approved CCR5 inhibitors), and characterization of other viral or drug resistance phenotypic properties to guide treatment.

Abstract: The invention provides fusion proteins comprising at least one fluorescent protein that is linked to at least one transporter protein that changes three-dimensional conformation upon specifically transporting its substrate. The transporter protein may be a nitrate transporter, a peptide transporter, or a hormone transporter. The invention provides fusion proteins comprising at least one fluorescent protein that is linked to at least one mechanosensitive ion channel protein. The invention also provides for methods of using the fusion proteins of the present invention and nucleic acids encoding the fusion proteins.

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: The invention relates in a first embodiment to a method for increasing the yield of replication-incompetent adenoviruses having at least a partial deletion in the E1-region, wherein said adenoviruses are generated in a production cell, the method comprising the steps of: (a) expressing in said production cell an adenoviral pIX polypeptide from a nucleic acid sequence encoding said adenoviral pIX polypeptide under the control of (i) at least a minimal endogenous pIX promoter and a heterologous promoter; or (ii) a heterologous promoter; and (b) expressing in said production cell the elements necessary for the production and assembly of said adenoviruses from corresponding coding sequences, thereby increasing the yield of said adenoviruses generated in said production cell in comparison to the yield of replication-incompetent adenoviruses having at least a partial deletion in the E1-region generated in said production cell in the absence of said nucleic acid sequence encoding said adenoviral pIX polypeptide.

Abstract: The present invention provides methods of achieving directed evolution of viruses by in vivo screening or “panning” to identify viruses comprising scrambled AAV capsids having characteristics of interest, e.g., tropism profile and/or neutralization profile (e.g., ability to evade neutralizing antibodies). The invention also provides scrambled AAV capsids and virus particles comprising the same.

Abstract: The present disclosure provides compositions and methods for the generation of an antibody or immunogenic composition, such as a vaccine, through epitope focusing by variable effective antigen surface concentration. Generally, the composition and methods of the disclosure comprise three steps: a “design process” comprising one or more in silico bioinformatics steps to select and generate a library of potential antigens for use in the immunogenic composition; a “formulation process”, comprising in vitro testing of potential antigens, using various biochemical assays, and further combining two or more antigens to generate one or more immunogenic compositions; and an “administering” step, whereby the immunogenic composition is administered to a host animal, immune cell, subject or patient. Further steps may also be included, such as the isolation and production of antibodies raised by host immune response to the immunogenic composition.

Abstract: The present disclosure provides, inter alia, genetically encoded recombinant peptide biosensors comprising analyte-binding framework portions and signaling portions, wherein the signaling portions are present within the framework portions at sites or amino acid positions that undergo a conformational change upon interaction of the framework portion with an analyte.

Abstract: The present invention relates to polypeptide fragments comprising an amino-terminal fragment of the PA subunit of a viral RNA-dependent RNA polymerase or variants thereof possessing endonuclease activity, wherein said PA subunit is from a virus belonging to the Orthomyxoviridae family. This invention also relates to (i) crystals of the polypeptide fragments which are suitable for structure determination of said polypeptide fragments using X-ray crystallography and (ii) computational methods using the structural coordinates of said polypeptide to screen for and design compounds that modulate, preferably inhibit the endonucleolytically active site within the polypeptide fragment. In addition, this invention relates to methods identifying compounds that bind to the PA polypeptide fragments possessing endonuclease activity and preferably inhibit said endonucleolytic activity, preferably in a high throughput setting.

Abstract: A leucine zipper variant, a polynucleotide encoding the leucine zipper variant, a method of preparing a leucine zipper variant, a method of inhibiting HDM2- and/or HDMX using the leucine zipper variant, and a method of the prevention and/or treatment of cancer using the leucine zipper variant.

Abstract: The present invention relates to a polypeptide that is capable of inhibiting transcription and expression of fatty acid synthase (FAS) and the polynucleotides encoding therefor, as well as the use thereof. Specifically, the present invention relates to a polypeptide that can inhibit the transcription and expression of FAS at the molecular level, the cellular level and in vivo, and can therefore prevent the overexpression of FAS. Said polypeptide and related peptidomimetics, including functional fragments or functional varieties thereof, and the genes encoding therefor, can be widely used in preventing and treating tumors such as liver cancer, and diseases closely related to the metabolism of fatty acid synthase, such as fatty liver and obesity.

Abstract: The present invention relates to Huwentoxin-IV variants, polynucleotides encoding them, methods of making and using the foregoing, and methods of alleviating pain with peptide inhibitors of Nav1.7.

Abstract: A system and method of adapting host cells to suspension cell culture and suspension cell lines ATCC PTA-12593 and ATCC PTA-12461 produced thereby are disclosed. The method includes the serial replating of substantially undiluted culture cells onto a surface area until cell clumps are visualized and then, upon cell clumping, moving the cells into a suspension culture system.

Abstract: The present invention is related to a method to reduce peptide amidation activity in a given cell line, cell lines with reduced peptide amidation activity, and uses thereof.

Abstract: Novel transcription units that may be used in expression vectors. The transcription unit allow antibodies to be produced whose gain in productivity is not linked to a particular antigenic target antibody and therefore by extrapolation to a given recombinant protein, nor linked to the culture medium.

Abstract: The present invention relates to polypeptides and their uses as apelin inhibitors. More particularly, the present invention relates to a polypeptide comprising the sequence as set forth in SEQ ID NO:1 wherein at least one arginine residue at position 18, 19, 22 or 23 has been substituted or deleted.

Abstract: The present disclosure provides engineered transaminase polypeptides for the production of amines, polynucleotides encoding the engineered transaminases, host cells capable of expressing the engineered transaminases, and methods of using the engineered transaminases to prepare compounds useful in the production of active pharmaceutical agents. The present disclosure provides engineered polypeptides having transaminase activity, polynucleotides encoding the polypeptides, methods of the making the polypeptides, and methods of using the polypeptides for the biocatalytic conversion of ketone substrates to amine products. The present enzymes have been engineered to have one or more residue differences as compared to the amino acid sequence of the naturally occurring transaminase of Vibrio fluvialis. In particular, the transaminases of the present disclosure have been engineered for efficient formation of chiral tryptamine derivatives from its corresponding prochiral ketone substrates.

Abstract: The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.

Abstract: Improved compositions and methods for AAV mediated gene therapy are disclosed.

Abstract: Drug compositions, fusions and conjugates are provided. The drug fusions and conjugates contain a therapeutic or diagnostic agent that is fused or conjugated to an antigen-binding fragment of an antibody that binds serum albumin. The drug compositions, fusions and conjugates have a longer in vivo half-life in comparison with the unconjugated or unfused therapeutic or diagnostic agent.

Abstract: The present invention relates to peptidomimetics that mimic helix ?B of the C-terminal transactivation domain of HIF-1?. Methods of using the peptidomimetics to, e.g., inhibit the HIF-1?-p300/CBP interaction, are also disclosed.

Abstract: The present invention relates to a one-vector expression system comprising a sequence encoding two polypeptides, such as tyrosine hydroxylase (TH) and GTP-cyclohydrolase 1 (GCH1). The two polypeptides can be should preferentially be expressed at a ratio between 3:1 and 15:1, such as between 3:1 and 7:1. The invention is useful in the treatment of catecholamine deficient disorders, such as dopamine deficient disorders including but not limited to Parkinson's Disease. Moreover, the present invention provides a method to deliver the vector construct in order to limit the increased production of the catecholamine to the cells in need thereof.

Abstract: The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology. Among other things, the present invention provides methods and compositions for targeting lysosomal enzymes to lysosomes using furin-resistant lysosomal targeting peptides. The present invention also provides methods and compositions for targeting lysosomal enzymes to lysosomes using a lysosomal targeting peptide that has reduced or diminished binding affinity for the insulin receptor.

Abstract: The present invention provides native Goodpasture antigen binding protein isoforms, monoclonal antibodies directed against such proteins, and methods for their use.

Abstract: Targeted therapeutics that localize to a specific subcellular compartment such as the lysosome are provided. The targeted therapeutics include a therapeutic agent and a targeting moiety that binds a receptor on an exterior surface of the cell, permitting proper subcellular localization of the targeted therapeutic upon internalization of the receptor. Nucleic acids, cells, and methods relating to the practice of the invention are also provided.

Abstract: An object of the present invention is to provide DNA which regulates the expression of miR-140, a reporter vector which contains the DNA, cells and animals into which the reporter vector is introduced and a screening system for drugs which control the expression of miR-140 and is also to contribute in the development of new therapeutic agents for cartilage diseases such as osteoarthritis and intervertebral disk degeneration using the screening system. The DNA sequence according to the present invention is able to express any gene in a site where miR-140 is expressed and, in addition, it is also able to be utilized for screening a drug which regulates the expression of miR-140. Moreover, the drug that is selected by the screening system of the present invention is expected as a therapeutic agent for cartilage diseases accompanied by abnormality of cartilage.

Abstract: The present invention relates to mutated tissue plasminogen activators, and their use for treating thrombotic diseases.

Abstract: The various embodiments herein provide a method for detecting the cancerous cells using the carbon nanotubes. The method comprises preparing a solution of the tissue cells. The prepared solution of the tissue cells is poured on a fabricated substrate to carry out an entrapment of the tissue cells on the substrate. The substrate is dried after the entrapment in an air ambient and observed under a scanning electron microscope. The cancer cell is detected based on the biomechanical properties such as softness, deformability and an elasticity of the cancer cells. The cancer cell is detected based on the deflection of the substrate due to the entrapment of the cancer cells.

Abstract: The present invention relates to an improved method for the serum-free production of an immortalized human cell line stably transfected under serum-free conditions with a specific vector carrying the gene coding for the protein of interest. Furthermore the invention relates to a production cell line obtained by said method, a production method for said protein of interest utilizing said production cell line, and the specific vector carrying the gene of interest itself.

Abstract: The present invention relates generally to the use of recombinant adeno-associated viruses (rAAV) for gene delivery and more specifically to the use of rAAV to deliver genes encoding human immunodeficiency virus entry inhibitors to target cells in mammals.

Abstract: Disclosed herein are AAV-based viral vectors encoding GNE from muscle-specific and non-muscle specific promoters, and the use of same in treating myopathies associated with altered GNE function.

Abstract: The present invention relates to a novel Listeria bacteriophage designated ProCC P825. In particular, the present invention relates to the endolysin PlyP825 encoded by the novel phage ProCC P825 and uses of the novel endolysin PlyP825 for controlling Listeria contamination and infection.