Abstract: The invention provides methods of inducing or improving responsiveness to a VEGF antagonist to a subject or a subject population comprising administering an adenovirus comprising a nucleic acid construct comprising a FAS-chimera gene operably linked to an endothelial cell-specific promoter and administering the VEGF antagonist.

Abstract: Provided are transgenic Caenorhabditis elegans (C. elegans) overexpressing DNA methyltransferase 3a (Dnmt3a) and a method of producing the same. According to the method, a specific mechanism and related factors for DNA methylation mediated by Dnmt3a may be found, and a critical gene for regulating the life span of C. elegans may be identified, and therefore C. elegans may be used as an animal model for screening a drug for a DNA methylation-related disease.

Abstract: Methods and compositions for reducing expression of genes on Chromosome 21 (“Chr 21”) by targeting an XIST transgene to the Dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) gene or a Regulator of calcineurin 1 (RCAN1) gene, and cells and transgenic animals comprising an XIST transgene inserted into a DYRK1A or RCAN1 allele, e.g., cells and animals trisomic for human Chr 21 and mouse Chr 16.

Abstract: Compositions for a phage particle are disclosed. The phage particle is non-replicating and includes at least one heterologous nucleic acid sequence that is capable of being expressed in a target bacteria. The expressed heterologous nucleic acid sequence is non-lethal to the target bacteria.

Abstract: A method for predicting a subject's response to a TUSC2 therapy is provided. In particular, a subject's response is predicted based on the proportion of cancers cells that are apoptotic. Also provided is a method of treating a subject previously predicted to have a favorable response with a TUSC2 therapy. Methods for treating cancer by administration of a TUSC2 therapeutic in conjunction with an EGFR inhibitor and/or a protein kinase inhibitor are also disclosed. Kits and reagents for use in TUSC2 therapy are provided.

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a mRNA solution and a lipid solution, wherein the mRNA solution and/or the lipid solution are at a pre-determined temperature greater than ambient temperature.

Abstract: Provided are methods of diagnosing IgA nephropathy in a subject. Optionally, the methods comprise isolating an IgG from the subject and determining whether the IgG binds to a galactose-deficient IgA1. Optionally, the methods comprise providing a biological sample from the subject and detecting in the sample a mutation in a IGH gene, wherein the mutation is in a nucleotide sequence encoding a complementarity determining region 3 (CDR3) of a IGH variable region. Optionally, the methods comprise determining a level of IgG specific for a galactose-deficient IgA1 in the subject. Also provided are methods of treating or reducing the risk of developing IgA nephropathy in a subject.

Abstract: A 3D organotypic culture which phenocopies aggressive, invasive cancer and methods of use thereof are provided.

Abstract: The present invention is directed to (the use of) a solution containing at least one nucleic acid (sequence) and free mannose for lyophilization, transfection and/or injection, particularly of RNA and mRNA. The inventive solution exhibits a positive effect on stabilization of the nucleic acid (sequence) during lyophilization and storage but also leads to a considerable increase of the transfection efficiency of a nucleic acid. It thus also increases in vivo expression of a protein encoded by such a nucleic acid upon increased transfection rate. The present invention is furthermore directed to a method of lyophilization using the mannose-containing solution, to pharmaceutical compositions, vaccines, kits, first and second medical uses applying such a mannose-containing solution and/or a nucleic acid (sequence) lyophilized or resuspended with such a solution.

Abstract: Described are means and methods for removing a proteolytic cleavage site from a protein, the method comprising providing a cell that expresses pre-mRNA encoding the protein with an anti-sense oligonucleotide that induces skipping of the exonic sequence that encodes the proteolytic cleavage site, and allowing translation of mRNA produced from the pre-mRNA.

Abstract: Compositions and methods for increasing or decreasing DNA repair are provided. Composition includes a Brd4 polypeptide, fragment, fusion, or variant thereof. The fusion protein can include a protein transduction, a targeting domain, or a combination thereof to enhance delivery of the fusion protein to the interior of a particular target cell, such as a cancer cell. Inhibitory nucleic acids that target a Brd4 mRNA and antibodies that target a Brd4 polypeptide are also disclosed. The inhibitory nucleic acid or antibody can target a sequence or epitope on Brd4 isoform B that is absent on Brd4 isoform A and Brd4 isoform C. Methods for increasing or decreasing the sensitivity of cells to a DNA damaging agent, methods of treating cancer, and methods of determining cells' sensitivity to a DNA damaging agent are also disclosed.

Abstract: Provided herein are mitochondrial-nuclear exchanged cells and animals comprising mitochondrial DNA (mtDNA) from one subject and nuclear DNA (nDNA) from a different subject. Methods for producing a mitochondrial-nuclear exchanged animal and animals made by the methods are provided. Also provided are methods of screening for agents useful for treating a disease or disorder using mitochondrial-nuclear exchanged animals or cells, tissues or organs thereof.

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: This invention relates to a compound that induces or activates telomerase activity based on the nucleotide sequence of the GSE 24.2 fragment of dyskerin or the protein or peptide sequence encoded by said nucleotide sequence. Another part of the invention relates to vectors that comprise said sequence and cells transformed thereby, and pharmaceutical compositions that contain all these elements. These compositions may be used in the treatment of diseases from the following group: ageing or acceleration of ageing, neurodegenerative diseases and dyskeratosis congenita.

Abstract: Compositions and methods of using cells derived from umbilical cord tissue, to stimulate and support angiogenesis, to improve blood flow, to regenerate, repair, and improve skeletal muscle damaged by a peripheral ischemic event, and to protect skeletal muscle from ischemic damage in peripheral vascular disease patients are disclosed. In particular, methods of treating a patient having a peripheral vascular disease by systemic administration of umbilical derived cells are disclosed.

Abstract: The present invention involves the preparation of a pharmaceutical liposomal system in an aqueous solution which contains a substance or polymer with mucomimetic and/or mucoadhesive properties. Due to the components and characteristics of the formulation described in this present patent it can be used as a precorneal tear film substitute. This invention is included in the pharmacy and medicine areas.

Abstract: This invention relates generally to a codon optimized nucleic acid encoding a retinitis pigmentosa GTPase regulator (RPGR) protein. The nucleic acid has enhanced stability during plasmid production relative to a wildtype cDNA encoding the RPGR protein. The invention also relates to expression cassettes, vectors, and host cells comprising the codon optimized nucleic acid. Methods for preparing a recombinant adeno-associated (rAAV) expression vector comprising the codon optimized nucleic acid sequence are also provided. The nucleic acids, expression cassettes, vectors, and host cells provided may be useful in the large scale production of rAAV expression vectors for gene therapy applications.

Abstract: Provided is a method of transfecting cells of the cochlea with an agent by electroporation, and in certain embodiments using a cochlear implant to provide at least one electroporation electrode.

Abstract: The present invention provides methods to promote the differentiation of pluripotent stem cells into insulin producing cells. In particular, the present invention provides a method to produce a population of cells, wherein greater than 85% of the cells in the population express markers characteristic of the definitive endoderm lineage.

Abstract: The present disclosure discloses novel Cry1Ac mutants with improved insecticidal activity and/or spectrum against pests belong to Order lepidoptera. Also provided are transgenic plants expressing the present protein and methods for controlling Lepidopteran pest using the present mutants.