Abstract: The present invention provides a transgenic mouse which comprises a deficiency for murine T lymphocytes, B lymphocytes and NK cells, a deficiency for murine MHC class I and MHC class II molecules, and a functional xenogenic SIRP? transgene. This mouse is useful for in vivo screening of various compounds, including immuno-therapeutic agents and vaccines. The said mouse is also useful for testing the in vivo metabolism of xenobiotic compounds.

Abstract: The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3?UTR element comprising a nucleic acid sequence which is derived from the 3?UTR of an albumin gene or from a variant of the 3?UTR of an albumin gene. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, the invention relates to the use of a 3?UTR element comprising a nucleic acid sequence which is derived from the 3?UTR of an albumin gene or from a variant of the 3?UTR of an albumin gene for the stabilization and/or prolongation of protein expression from a nucleic acid sequence comprising such 3?UTR element.

Abstract: Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of a lysosomal storage disease.

Abstract: The present invention is directed to the use of mitotically and/or lethally inactivated stem cells for the repair of damaged organs and/or tissues. Stem cells are mitotically and/or lethally inactivated and transplanted into damaged tissue. Any form of ex vivo inactivation of stem cells may be used such that the stem cells cannot undergo mitosis or cell division before in vivo application. Mitotically and/or lethally inactivated stem may be used to ameliorate numerous disease, injury, traumatic, ischemic, aging, and/or degenerative conditions in different types of organs and/or tissues.

Abstract: Genetically modified mice are provided that express human ? variable (hV?) sequences, including mice that express hV? sequences from an endogenous mouse ? light chain locus, mice that express hV? sequences from an endogenous mouse ? light chain locus, and mice that express hV? sequences from a transgene or an episome wherein the hV? sequence is linked to a mouse constant sequence. Mice are provided that are a source of somatically mutated human ? variable sequences useful for making antigen-binding proteins. Compositions and methods for making antigen-binding proteins that comprise human ? variable sequences, including human antibodies, are provided.

Abstract: Genetically modified mice are provided that express human ? variable (hV?) sequences, including mice that express hV? sequences from an endogenous mouse ? light chain locus, mice that express hV? sequences from an endogenous mouse ? light chain locus, and mice that express hV? sequences from a transgene or an episome wherein the hV? sequence is linked to a mouse constant sequence. Mice are provided that are a source of somatically mutated human ? variable sequences useful for making antigen-binding proteins. Compositions and methods for making antigen-binding proteins that comprise human ? variable sequences, including human antibodies, are provided.

Abstract: Methods and compositions are provided for delivery of a polynucleotide encoding a gene of interest, typically an antigen, to a dendritic cell (DC). The virus envelope comprises a DC-SIGN specific targeting molecule. The methods and related compositions can be used to treat patients suffering from a wide range of conditions, including infection, such as HIV/AIDS, and various types of cancers.

Abstract: The invention relates generally to compositions and methods of using transgenic non-human animals expressing human SIRP? that are engrafted with a human hematopoietic system. In various embodiments, the human hematopoietic system engrafted, human SIRP? transgenic non-human animals of the invention are useful as systems for the in vivo evaluation of the growth and differentiation of hematopoietic and immune cells, for the in vivo assessment of an immune response, for the in vivo evaluation of vaccines and vaccination regimens, for in vivo production and collection of immune mediators, including human antibodies, and for use in testing the effect of agents that modulate hematopoietic and immune cell function.

Abstract: Disclosed herein are methods, compositions, and kits for high efficiency, site-specific genomic editing of cells.

Abstract: The present invention relates to the development and manufacturing of viral vaccines. In particular, the invention relates to the field of industrial production of viral vectors and vaccines, more in particular to the use of avian embryonic stem cells, preferably the EBx® cell line derived from duck embryonic stem cells, for the production of viral vectors and viruses. The invention is particularly useful for the industrial production of viral vaccines to prevent viral infection of humans and animals.

Abstract: Described herein are synthetic, modified RNAs for changing the phenotype of a cell, such as expressing a polypeptide or altering the developmental potential. Accordingly, provided herein are compositions, methods, and kits comprising synthetic, modified RNAs for changing the phenotype of a cell or cells. These methods, compositions, and kits comprising synthetic, modified RNAs can be used either to express a desired protein in a cell or tissue, or to change the differentiated phenotype of a cell to that of another, desired cell type.

Abstract: An obese mouse model was developed by overexpressing the mitochondrial protein prohibitin (PHB) in white adipose tissue (WAT) specific manner driven by adipocyte protein 2 (aP2) promoter. These mice begin to develop obesity as a result of mitochondrial remodeling (upregulation of mitochondrial biogenesis and function) in WAT.

Abstract: Methods for obtaining multipotent Apelin receptor-positive lateral plate mesoderm cells, mesenchymal stem cells, and mesangioblasts under serum-free conditions are disclosed.

Abstract: The invention is directed to in vitro methods of inducing differentiation of anterior foregut endoderm and the enriched populations of anterior foregut endoderm produced by such methods. Such enriched populations are useful for studies of the molecular events that occur during differentiation and for generating cells for cell replacement therapy.

Abstract: Genetically modified non-human animals and methods and compositions for making and using them are provided, wherein the genetic modification comprises a deletion of the endogenous low affinity Fc?R locus, and wherein the mouse is capable of expressing a functional FcR?-chain. Genetically modified mice are described, including mice that express low affinity human Fc?R genes from the endogenous Fc?R locus, and wherein the mice comprise a functional FcR?-chain. Genetically modified mice that express up to five low affinity human Fc?R genes on accessory cells of the host immune system are provided.

Abstract: The present invention relates to testis somatic cell-derived pluripotent stem cells, and more particularly, to pluripotent adult stem cells which exhibit a positive immune reaction to both CD34 and CD73 and which are derived from testis somatic cells. The present invention further relates to a method for producing the testis somatic cell-derived pluripotent stem cells, and to a pharmaceutical composition including same for the treatment of erectile dysfunction.

Abstract: Provided are compositions and methods that involve cancer cells which are modified so that they can form orthotopic tumors in a non-human mammal, and wherein metastasis of the tumor can be controlled. The cancer cells, which may be human cancer cells, are modified so that expression of a human chemokine receptor can be modulated. Modulating expression of the human chemokine receptor allows selective initiation of metastasis. Kits which contain the modified cancer cells are provided. A method for identifying agents which can inhibit metastasis using non-human mammals having orthotopic tumors formed using the modified cancer cells is also included.

Abstract: A sustained culture of isolated avian gonocytes is provided, as well as a method of making and using the same. A chimeric avian containing an isolated gonocyte and a transgenic avian produced using the chimeric avian are also provided. The cell and method may be employed to make, among other things, transgenic avian that produce a heterologous protein, e.g., a therapeutic protein.

Abstract: The invention provides compositions and methods useful to prepare segmented, negative strand RNA viruses, e.g., orthomyxoviruses such as influenza A viruses, entirely from cloned cDNAs and in the absence of helper virus.

Abstract: The invention provides transgenomic mitochondria, transmitochondrial cells and organisms, and the materials and methods for making such mitochondria, cells, and organisms.