Abstract: The present disclosure provides compositions and methods for the prevention or treatment of ocular neovascularization, such as AMD, in a human subject, by administering subretinally a pharmaceutical composition comprising a pharmaceutically effective amount of a vector comprising a nucleic acid encoding soluble Fms-related tyrosine kinase-1 (sFlt-1) protein to the human subject.

Abstract: The present invention relates to a drug carrier having L-DNA nanocage structure prepared by using L-DNA, the mirror form of natural DNA, as a backbone. The drug carrier of the present invention has very superior cellular uptake efficiency and serum stability, so that it can be applied to deliver various drugs into cells usefully.

Abstract: Provided are a diagnosis marker, a diagnosis method, and a therapeutic agent suitable for diagnosing and treating amyotrophic lateral sclerosis (ALS). Also provided are an animal model and a cell model suitable for developing a therapeutic agent and a treatment method for ALS. The diagnosis method for ALS includes: an isolation step in which a nucleic acid is isolated from a specimen taken from a subject; a detection step in which bases expressed in a human chromosome 10 optineurin (OPTN) gene region are detected from the isolated nucleic acid; and a determination step in which it is determined whether or not the detected bases are mutated.

Abstract: In various embodiments, the present invention describes materials and methods for the local reprogramming of cells in a location where the treatment is applied. The invention can be used to replace lost cells or to restore function to tissue damaged due to disease, injury or genetic defect. In various embodiments, the treatment includes a semisolid hydrogel embedded with liposomes. The liposomes can contain an effector molecule or molecules. When phagocytic cells such as monocytes infiltrate the hydrogel, they encounter the liposomes and incorporate the liposomes carrying the effector molecules into the cells. In some embodiments, the effector molecules can induce angiogenesis. The matrix can contain other effector molecules designed to attract specific cells to the matrix. The cells can also remain in the matrix and secret molecules such as proteins and hormones that will diffuse through the matrix material to the surrounding tissue.

Abstract: A method that includes bringing a nuclear reprogramming substance (DLX4 gene, OCT3/4 gene, and SOX2 gene) into contact with a cell and thereby producing iPS cells.

Abstract: An isolated nucleic acid molecule comprising the nucleic acid sequence of SEQ ID NO:1, or a nucleic acid sequence of at least 1000 bp having at least 70% identity to the sequence of SEQ ID NO:1. The isolated nucleic acid molecule can lead to the expression of a gene in retinal ON bipolar cells when a nucleic acid sequence coding for a gene is operatively linked to the isolated nucleic acid molecule.

Abstract: Aspects of the present invention are drawn to compositions of somatic cells with innate potential for pluripotency (SCIPP). SCIPP have the capacity to differentiate into functional derivatives of each of the major germ layers (i.e., ectodermal, endodermal and mesodermal). Also provided are methods and kits for identifying and isolating the somatic cells from a subject as well as for employing SCIPP for research or therapeutic purposes.

Abstract: This invention is intended to develop a promoter that can strongly induce marker gene expression throughout an embryo, so as to simply, efficiently, and accurately identify a transgenic insect at an early developmental stage, and to provide a gene expression vector into which such promoter has been incorporated as a transformant discrimination marker. Such exogenous gene expression vector comprises a polynucleotide comprising the nucleotide sequence as shown in SEQ ID NO: 1 as a promoter.

Abstract: Genetically modified non-human animals are provided that express an immunoglobulin variable domain that comprises at least one histidine, wherein the at least one histidine is encoded by a substitution of a non-histidine codon in the germline of the animal with a hisidine codon, or the insertion of a histidine codon in a germline immunoglobulin nucleic acid sequence. Immunoglobulin genes comprising histidines in one or more CDRs, in an N-terminal region, and or in a loop 4 region are also provided. Immunoglobulin variable domains comprising one or more histidines (e.g., histidine clusters) substituted for non-antigen-binding non-histidine residues. Non-human animals that are progeny of animals comprising modified heavy chain variable loci (V, D, J segments), modified light chain variable loci (V, J segments), and rearranged germline light chain genes (VJ sequences) are also provided. Non-human animals that make immunoglobulin domains that bind antigens in a pH-sensitive manner are provided.

Abstract: Methods are disclosed herein for increasing bone mass and strength or bone fracture healing in a subject. The methods include administering to the subject a therapeutically effective amount of multipotent stem cells, wherein each multipotent stem cell is transformed with a recombinant nucleic acid molecule comprising a heterologous promoter operably linked to a nucleic acid encoding platelet derived growth factor (PDGF) B, and wherein the multipotent stem cells express a sufficient amount of PDGFB to increase bone mass and strength or bone fracture healing. A lentiviral vector also is disclosed that includes a phosphoglycerate kinase-1 (PGK) promoter operably linked to a nucleic acid encoding PDGFB.

Abstract: A human artificial chromosome vector comprising a gene encoding a human antibody heavy chain, a gene encoding a human antibody light chain, and a gene encoding an IgM heavy chain constant region derived from a nonhuman animal.

Abstract: The present invention relates to mutants of Cre recombinase.

Abstract: Disclosed are genetically modified nudiviruses capable of being sexually transmitted by an insect useful for controlling pest populations. The genetically modified nudiviruses are capable of causing sterility in a target population of insects. Also disclosed are insects infected with the disclosed genetically modified nudiviruses, methods of making the genetically modified nudiviruses, and methods of using the genetically modified nudiviruses to control an insect pest population.

Abstract: Recombinant vectors in which expression of one or more elements (e.g. genes required for viral replication, detectable imaging agents, therapeutic agents, etc.) is driven by a truncated CCN 1 cancer selective promoter (tCCN1-Prom) are provided, as are cells and transgenic animals that contain such vectors. The vectors are used in cancer therapy and/or diagnostics, and the transgenic mice are used to monitor cancer progression, e.g. in screening assays.

Abstract: The instant invention provides polyvalent RNA nanoparticles comprising RNA motifs as building blocks that can form RNA nanotubes. The polyvalent RNA nanoparticles are suitable for therapeutic or diagnostic use in a number of diseases or disorders.

Abstract: Methods for treating inflammation are disclosed, such as for treating ocular inflammation. In some embodiments, the ocular inflammation is inflammation of an ocular surface, such as keratitis. The methods include administering to a subject with inflammation a therapeutically effective amount of SLURP1, or a nucleic acid encoding SLURP1, thereby treating the inflammation.

Abstract: The vast differentiation potential of human embryonic and induced pluripotent stem cells, including their potential to cascade through all of the somatic cell lineages and to display the complete transcriptional regulatory network of human biology, has generated interest in deriving scalable, purified, and identified cell types and methods of discovering the precise structure of the human regulatory network. However, the innate capacity of pluripotent cells to display all these lineages is not necessarily reflected during their culture in vitro. The clonal isolation and propagation of progenitors greatly facilitates the generation of highly purified and identified formulations for research and therapeutic purposes.

Abstract: The present invention concerns phase changing injectable formulations for organ augmentation containing active agents, such as bioactive cell populations, and methods of making and using the same.

Abstract: Correlations between polymorphisms and breast cancer are provided. Methods of diagnosing, prognosing, and treating breast cancer are provided. Systems and kits for diagnosis, prognosis and treatment of breast cancer are provided. Methods of identifying breast cancer modulators are also described.

Abstract: The present invention relates to fibrinogen preparations enriched in ?-extended fibrinogen. Compositions comprising such preparations show improved clotting properties compared to preparations based on HMW Fib which typically contain no or only low amounts of ?-extended fibrinogen. In particular, clot formation time and the clot strength of a clot made by ?-extended fibrinogen are improved. In addition, plasmin-mediated degradation of ?-extended fibrinogen is reduced as compared to plasma derived fibrinogen.