Abstract: The present invention relates generally to the field of tissue engineering and in particular to the production of tissue films or cell matrices, which can be used as a living tissue substitute or an artificial tissue construct in tissue repair or replacement.

Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harbouring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.2991+1655 A>G CEP290 mRNA.

Abstract: The invention provides a method of treating a vascular condition within a subject comprising administering a therapeutic agent to said subject that is capable of modulating the expression levels of a long non-coding RNA (IncRNA) selected from the group consisting of lncRNA2, lncRNA4, lncRNA5, lncRNA6, ncRNA7 and ncRNA8 as defined in FIG. 39.

Abstract: Methods and compositions are provided for modulating, e.g., reducing, viral coding sequence expression in mammals and mammalian cells. In the subject methods, an effective amount of an RNAi agent, e.g., an interfering ribonucleic acid (such as an siRNA or shRNA) or a transcription template thereof, e.g., a DNA encoding an shRNA, is introduced into a target cell, e.g., by being administered to a mammal that includes the target cell, e.g., via a hydrodynamic administration protocol. Also provided are RNAi agent pharmaceutical preparations for use in the subject methods. The subject methods and compositions find use in a variety of different applications, including academic and therapeutic applications.

Abstract: Provided herein are isolated populations of kidney cells harvested from differentiated cells of the kidney, wherein the cells have been expanded in vitro. The kidney cells preferably produce erythropoietin (EPO). The kidney cells may also be selected based upon EPO production. Methods of producing an isolated population of EPO producing cells are also provided, and methods of treating a kidney disease resulting in decreased EPO production in a patient in need thereof are provided, including administering the population to the patient, whereby the cells express EPO in vivo in an oxygen tension-dependent manner.

Abstract: The instant disclosure features single-stranded RNA molecules comprising one or more internal, non-nucleotide spacers. A non-nucleotide spacer covalently links nucleotide portions of the molecule. The single-stranded RNA molecules function as guide or antisense strands that are capable of inhibiting gene expression via an RNA interference mechanism and, thus, represent single-stranded RNAi agents. The single-stranded RNAi molecules can be used in methods for a variety of therapeutic, diagnostic, target validation, genomic discovery, genetic engineering, and pharmacogenomic applications.

Abstract: Androgen receptor-based vaccines for eliciting an immune reaction in vivo against cells expressing androgen receptor are disclosed. The vaccines are useful in the treatment of prostate cancer. Also disclosed are methods for inducing immune reaction to androgen receptor or treating prostate cancer in a mammal, using the vaccines and pharmaceutical compositions comprising the vaccines.

Abstract: This invention provides a method for the in vivo delivery of oligonucleotides. The invention utilizes the presence of one or plurality of HES linked to an oligonucleotide to deliver a nucleic acid sequence of interest into the cytoplasm of cells and tissues of live organisms. The delivery vehicle is nontoxic to cells and organisms. Since delivery is sequence-independent and crosses membranes in a receptor-independent manner, the delivered oligonucleotide can target complementary sequences in the cytoplasm as well as in the nucleus of live cells. Sequences of bacterial or viral origin can also be targeted. The method can be used for delivery of genes coding for expression of specific proteins, antisense oligonucleotides, siRNAs, shRNAs, Dicer substrates, miRNAs, anti-miRNAs or any nucleic acid sequence in a living organism. The latter include mammals, plants, and microorganisms such as bacteria, protozoa, and viruses.

Abstract: Provided are a therapeutic and/or prophylactic agent for a lung disease and a method for screening for the therapeutic and/or prophylactic agent. Provided are a therapeutic and/or prophylactic agent for a lung disease comprising an Arid5A inhibitor as an active ingredient and a method for screening for the therapeutic and/or prophylactic agent.

Abstract: Aspects of the present disclosure include methods that include co-culturing a cell and a microparticle that includes a capture ligand, in a culture medium under conditions in which a biomarker produced by the cell is bound by the capture ligand. Such methods may further include detecting (e.g., by flow or mass cytometry) complexes that include the microparticle, the capture ligand, the biomarker, and a detection reagent. The methods may further include determining the proportion or number of cells among a heterogeneous cell population that produced the biomarker and/or the level of biomarker secreted by such cells. Compositions, systems and kits are also provided.

Abstract: Compounds useful as labels with properties comparable to known fluorescent compounds. The compounds are conjugated to proteins and nucleic acids for biological imaging and analysis. Synthesis of the compounds, formation and use of the conjugated compounds, and specific non-limiting examples of each are provided.

Abstract: Embodiments of the present disclosure include particles, methods of making particles, methods of delivering an active agent using the particle, and the like.

Abstract: Systems and methods for generating a mask for automated assessment of embryo quality are disclosed herein. The method for generating a mask for automated assessment of embryo quality can include receiving an image, including a plurality of pixels, of a human embryo from an imaging system. A pixel can be selected and features of the selected pixel can be determined by generating a plurality of random boxes of random sizes and at random locations about the selected pixel. The selected pixel can be identified as one of: inside of a mask area; and outside of the mask area based on the determined features.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Alpha-L-Iduronidase (IDUA), in particular, by targeting natural antisense polynucleotides of Alpha-L-Iduronidase (IDUA). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of IDUA.

Abstract: The present invention provides a novel technique in a cell culture method using a cell culture vessel coated with a laminin fragment, which novel technique achieves cell culture as in the case of using a recommended coating concentration even when the coating concentration is lower than the recommended coating concentration. The present invention relates to a method for enhancing an activity for mammalian cultured cells of a laminin fragment or a variant thereof each having integrin binding activity.

Abstract: The present invention relates to compositions and methods of inhibiting stem cell binding to organs and tissues, including the blocking of stem cell binding to germinal centers present in lymph tissue. Disclosed are compositions and methods for regenerating germinal centers in lymphatic tissue. Included in the compositions are adjuvants, agonists to CD40, CD28 and the IL-21 receptor, and antagonist to CD20.

Abstract: The invention relates to novel targets for immune response modulation, treatment of tuberculosis infection and epitopes of Mycobacterium tuberculosis, or subsequences, portions or modifications thereof, and methods and compounds for treatment and prevention of tuberculosis infection.

Abstract: This invention relates, e.g., to an inhibitory peptide or CPP inhibitor which specifically binds to p53 having an aberrant conformation (e.g., is aggregated or misfolded) and inhibits p53 amyloidogenic aggregation or restores proper folding of the misfolded protein. Methods of using the inhibitory peptide or CPP inhibitor (e.g. to treat subjects having tumors that comprise aggregated p53) are described.

Abstract: There are provided, inter alia, methods and compositions to treat autoimmune disease including invasiveness of fibroblast-like synoviocytes in rheumatoid arthritis.

Abstract: Oligonucleotide compounds modulate expression and/or function of Erythropoietin (EPO) polynucleotides and encoded products thereof. Methods for treating diseases associated with Erythropoietin (EPO) comprise administering one or more oligonucleotide compounds designed to inhibit the EPO natural antisense transcript to patients.

Abstract: The present invention relates compounds for stabilizing cells and methods of their use.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of RNase H1, in particular, by targeting natural antisense polynucleotides of RNase H1. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of RNASE H1.

Abstract: The present disclosure relates to methods of treating heat stock factor 1 (HSF1)-related diseases such as cancer, autoimmune and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.

Abstract: The present invention comprises compositions, methods and devices for creating an infection-mimicking environment within a polymer scaffold to stimulate antigen-specific dendritic cell activation. Devices of the present invention are used to provide protective immunity to subjects against infection and cancer.

Abstract: Provided herein are bitter taste receptor ligands, related agents, combinations, compositions, methods and systems for modulating release of a metabolic hormone in vitro and in vivo from cells of the GI tract of an individual.

Abstract: Disclosed are methods of treating and/or inhibiting a viral infection in a subject. The methods include administering a therapeutically effective amount of heparin-binding peptide. Also disclosed herein are methods for blocking viral binding to a cell. Further disclosed are anti-viral compositions for administration to a subject infected with a virus. Administration of the anti-viral composition inhibits viral infection of the subject.

Abstract: Antimicrobial peptides represent a relatively new discovery in the immune system pathway. Recent designs of synthetically engineered antimicrobial peptides have demonstrated increased potency and efficacy/tolerability, enhanced specificity, and reduced toxicity in comparison. One such peptide, XYLENTRA®, has shown/demonstrated significant promise from significant in vitro studies against a large + pathogens. Additionally, extensive animal studies have shown that the XYLENTRA® is an antimicrobial peptide against a large number of pathogens. The XYLENTRA® peptide is also solute resistant. The peptide XYLENTRA® has shown/demonstrated significant antibacterial activity on test organisms Staphylococcus aureus MTCC 96 and Pseudomonas aeruginosa. MTCC741. A substantial decrease in the microbial population level was observed in animals treated with peptide using the protocol described in detail in the application.

Abstract: In one or more embodiments, the present invention provides iodine-functionalized phenylalanine-based poly(ester urea)s (PEUs) (and related methods for their synthesis and use) that are metal free, degradable, radiopaque and suitable for use in surgical implants and other medical devices used within a patient. In one or more embodiment of the present invention 4-Iodo-L-phenylalanine and L-phenylalanine are separately reacted with 1,6-hexanediol to produce two monomers, bis-4-I-L-phenylalanine-1,6-hexanediol-diester (1-IPHE-6 monomer) and bis-L-phenylalanine-1,6-hexanediol-diester (1-PHE-6 monomer). It has been found that by varying the feed ratio of the 1-IPHE-6 and 1-PHE-6 monomers, the copolymer composition may be modulated to predictably create phenylalanine-based PEUs having a wide variation in thermal, mechanical and radiopacity properties.

Abstract: The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.

Abstract: The invention relates to double-stranded ribonucleic acids (dsRNAs) targeting gene expression of phosphatidylinositol 4-kinase (PI4K), in particular human phosphatidylinositol 4-kinase, catalytic, beta polypeptide (PIK4CB) or human phosphatidylinositol 4-kinase, catalytic, alpha polypeptide (PIK4CA), and their use for treating infection by positive stranded RNA viruses such as hepatitis C virus (HCV). Each dsRNA comprises an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PIK4CB or PIK4CA target mRNA. A plurality of such dsRNA may be employed to provide therapeutic benefit. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, and including a delivery modality such as fully encapsulated liposomes or lipid complexes.

Abstract: The invention relates to nucleic acid modifications for a directed expression modulation by the targeted insertion or removal of CpG dinucleotides. The invention also relates to modified nucleic acids and expression vectors.

Abstract: The present invention comprises compositions, methods, and devices for creating an infection-mimicking environment within a polymer scaffold to stimulate antigen-specific dendritic cell activation. Devices of the present invention are used to provide protective immunity to subjects against infection and cancer.

Abstract: Methods and compositions relating to the production of induced pluripotent stem cells (iPS cells) are disclosed. For example, induced pluripotent stem cells may be generated from peripheral blood cells, such as human blood progenitor cells, using episomal reprogramming and feeder-free or xeno-free conditions. In certain embodiments, the invention provides novel methods for improving overall reprogramming efficiency with low number of blood progenitor cells.

Abstract: Described herein are conjugated modified oligonucleotides that are complementary to a target RNA. The conjugate facilitates cellular uptake of the modified oligonucleotide, resulting improved potency.

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: Compositions and methods described herein include somatic cells that are competent for reprogramming and malignant transformation and are characterized by a reduction of the levels of Sp100 in the cells, cells having markers of pluripotent stem cells, and methods for preparing same. Methods for reversably regulating aging or reprogramming to pluripotency in a somatic cell involve modulating the expression of Sp100 therein. Methods and compositions for retarding the growth of or suppressing unwanted cell proliferation involve expressing, inducing expression of, or upregulating, Sp100 in a targeted cell that is undergoing unrestricted proliferation or replication or increasing exposure to Sp100 in the environment or microenvironment of the targeted cell. Also disclosed are methods for treating a proliferative disease or condition by increasing expression or levels of Sp100 in the targeted cell or its environment.

Abstract: Methods and compositions involving exosomes or lipid nanovesicles are provided. For example, certain aspects relate to compositions comprising exosomes obtained from cells that have been induced to undergo oxidative stress or stimulated. Furthermore, some aspects of the invention provide methods of treating a subject at risk or having a demyelinating disorder using the compositions.

Abstract: In certain embodiments, the disclosure relates to the polynucleotide sequences of respiratory syncytial virus (RSV). In certain embodiments, the disclosure relates to isolated or recombinant nucleic acids and polypeptides comprising desirable nucleic acid sequences and mutations disclosed herein. In certain embodiments, isolated or recombinant RSV comprising the nucleic acids and polypeptides disclosed herein (e.g., attenuated recombinant RSV) are also provided, as are immunogenic compositions including such nucleic acids, polypeptides, and RSV genomes that are suitable for use as vaccines. Attenuated or killed RSV containing these nucleic acids and mutation in the form of copied nucleic acids (e.g., cDNAs) are also contemplated.

Abstract: The present invention provides a method for inducing cardiac differentiation of a pluripotent stem cell, which comprises the steps of (1) culturing a pluripotent stem cell in a medium containing a WNT signaling activator and a PKC activator and (2) culturing the cell after the step (1) in a medium containing a WNT signaling inhibitor, a Src inhibitor, and an EGFR inhibitor.

Abstract: Described herein are methods and compositions related to generation of induced pluripotent stem cells (iPSCs). Improved techniques for establishing highly efficient, reproducible reprogramming using non-integrating episomal plasmid vectors. Using the described reprogramming protocol, one is able to consistently reprogram non-T cells with close to 100% success from non-T cell or non-B cell sources. Further advantages include use of a defined reprogramming media E7 and using defined clinically compatible substrate recombinant human L-521. Generation of iPSCs from these blood cell sources allows for recapitulation of the entire genomic repertoire, preservation of genomic fidelity and enhanced genomic stability.

Abstract: The present application relates to compositions comprising selenium compounds, such as 5?-Methylselenoadenosine, a compound of Formula (I), and combinations thereof, and methods of using the same for inhibiting ? amyloid aggregation, ApoE4 expression, p38 or Tau protein phosphorylation, or increasing Neprilysin and Insulin Degrading Enzyme expression.

Abstract: The present disclosure relates to chimeric post-transcriptional regulatory elements (PRE) and vectors useful for expressing a protein in a cell. The PRE contains alpha, beta and optionally gamma subelements selected from different native PRE sequences and are discovered to be more potent than their native counterparts.

Abstract: A method of generating a particle is disclosed, the particle being for delivery of a polynucleotide to a target cell. The method comprises (a) contacting the polynucleotide with a composition comprising cationic molecules, wherein the cationic molecules condense the polynucleotide by electrostatic interactions to generate a complex, wherein the cationic molecules are not comprised in a liposome; and (b) covalently binding the complex to a targeting moiety at a pH equal to or below about 4.5, thereby generating the particle for delivery of the polynucleotide agent to the target cell. Use of the particles and compositions comprising same are also disclosed.

Abstract: Methods and compositions for modulating the activities of connexins are provided, including, for example, for use in post-surgical, trauma, or tissue engineering applications. These compounds and methods can be used therapeutically, for example, to reduce the severity of adverse effects associated diseases and disorders where localized disruption in direct cell-cell communication is desirable.

Abstract: The present invention relates to a process for purification of recombinant alpha-mannosidase, a process for production of alpha-mannosidase, a composition comprising alpha-mannosidase, use of the composition as a medicament, use as a medicament for the treatment of alpha-mannosidosis and a method of treating alpha-mannosidosis and/or alleviating the symptoms of alpha-mannosidosis.

Abstract: The disclosure provides a correlation between the expression level of the miR-193a gene, which can be regulated by its methylation status, and both tumorigenesis of and the resistance of a cancer cell to a pyrimidine antimetabolite (5-FU) based chemotherapy. In addition to the methylation status and the expression of miR-193a, its downstream genes, such as E2F1, SRSF2, and apoptotic genes such as caspase 2, are also involved and can serve as useful markers for cancer therapy prognosis and for therapy selection.

Abstract: Prodrugs or pharmaceutically acceptable salts, stereoisomers, solvates, or polymorphs thereof include a pharmaceutically and/or diagnostically active cationic steroidal antimicrobial (hereinafter “CSA”) compound or pharmaceutically acceptable salt, stereoisomer, solvate, or polymorph thereof, and one or more cleavable groups (C.G.). Some embodiments include a CSA compound prepared in an inactive or less active form and that is capable of conversion to a fully active form upon administration to a subject, upon preparation of a pharmaceutical formulation containing the CSA composition, and/or upon exposure to physiological conditions. Pharmaceutical compositions include the prodrug or pharmaceutically acceptable salt, stereoisomer, solvate, or polymorph thereof and one or more pharmaceutically acceptable excipients.

Abstract: Aspects of the present invention relate to peptides having anti-inflammatory activity, compositions containing one or more of the peptides, and use of the peptides to treat conditions associated with excessive inflammation in animals, particularly humans and other mammals.

Abstract: This invention relates to the use of CRISPR nucleic acids to screen for essential and non-essential genes and expendable genomic islands in bacteria, archaea, algae and/or yeast, to kill bacteria, archaea, algae and/or yeast, to identify the phenotype of a gene or genes, and/or to screen for reduced genome size and/or a gene deletion in bacteria, archaea, algae and/or yeast.

Abstract: Disclosed herein are antisense compounds and methods for decreasing CD40. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to CD40 include hyperproliferative disorders, graft versus host disease (GVHD), graft rejection, asthma, airway hyperresponsiveness, chronic obstructive pulmonary disease (COPD), multiple sclerosis (MS), systemic lupus erythematosus (SLE), and certain forms of arthritis.