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: A method of treating a cardiomyopathy in a subject includes administering directly to or expressing locally in a weakened, ischemic, and/or peri-infarct region of myocardial tissue of the subject an amount of SDF-1 effective to cause functional improvement in at least one of the following parameters: left ventricular volume, left ventricular area, left ventricular dimension, cardiac function, 6-minute walk test, or New York Heart Association (NYHA) functional classification.

Abstract: The present disclosure provides method of generating cardiomyocytes from post-natal fibroblasts. The present disclosure further provides cells and compositions for use in generating cardiomyocytes.

Abstract: The current invention discloses a genetically engineered bacterium used for the treatment of breast cancer. The said bacterium is attenuated Salmonella typhimurium VNP20009 with cloned L-methioninase gene. The method for constructing this genetically engineered bacterium and the application thereof are also disclosed herein. In the current invention, our biologic drug for the treatment of breast cancer is a type of safe, non-toxic new drug with anti-tumor activity. It can highly express methioninase through recombinant DNA technology using attenuated Salmonella typhimurium VNP20009 as a carrier, which has a strong anti-tumor activity and can meet the needs. The preparation method is simple and easy to operate, showing good application prospect.

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic stem cell.

Abstract: This disclosure relates to nanoparticles carrying nucleic acid cassettes for expressing RNA. In certain embodiments, the disclosure relates to improved methods for targeted delivery and expression of siRNAs in vivo using DNA-based siRNA-expressing nanocassettes and receptor-targeted nanoparticles. In certain embodiments, the disclosure relates to methods of targeted delivery of survivin siRNA expressing nanocassettes which enhance sensitivity of human cancer cells to anticancer agents.

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: The invention provides systems and methods for spatial sequestration of elements in nucleic acid circuits.

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: The usage of a stem cell in preparation of a tooth-like structure is provided. And a culture medium, a method for preparing an epithelial-like cell, a kit for preparing an ameloblast, a method for preparing an ameloblast are also provided. Specifically, the culture medium comprises a basal medium, which is DMEM/F12 medium; N2 supplement; retinoic acid; and BMP-4.

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

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: 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: Several embodiments disclosed herein relate generally to methods and compositions for the generation of biological pacemakers. In some embodiments, the methods comprise contacting non-pacemaker cells with one or more transcription factors (in vivo or in vitro) and inducing pacemaker functionality in the cells.

Abstract: Provided are a modified laminin having a cell-growth regulatory molecule bound to at least one site selected from the ? chain N-terminus, the ? chain C-terminus, the ? chain N-terminus and the ? chain N-terminus of laminin or a heterotrimeric laminin fragment, a method for culturing cells in the presence of the modified laminin, a method for establishing iPS cells in the presence of the modified laminin, and a culture substrate coated with the modified laminin. Human stem cells cultured in a xeno-free environment with the use of the modified laminin of the present invention can be provided as highly safe human stem cells applicable to regenerative medicine.

Abstract: The present invention relates to a therapeutic substance and/or medicament and methods relating to the use of said substance and/or medicament for skeletal muscle regeneration using mesenchymal stem cells (MSCs) which can be applied directly or shortly after muscle damage or injury.

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 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 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.