Abstract: The invention relates to a method of delivering exogenous DNA to a target cell of the mammalian central nervous system using an adeno-associated virus (AAV)-derived vector. Also included in the invention are the AAV-derived vectors containing exogenous DNA which encodes a protein or proteins which treat nervous system disease, and a method of treating such disease.

Abstract: Transgenic cells, transgenic mice having an Ikaros transgene and methods for the use thereof.

Abstract: In accordance with the present invention, there are provided CRF overproducing transgenic mice which exhibit endocrine abnormalities involving the hypothalamic-pituitary-adrenal axis, such as elevated plasma levels of ACTH and glucocorticoids. The transgenic mice of the present invention represent a genetic model of CRF overproduction, providing a valuable tool for investigating the long term effects of CRF excess and dysregulation in the central nervous system.

Abstract: A targeted disruption of the NF1 gene in mice has been used to demonstrate that both neural crest- and placode-derived sensory neurons isolated from NF1(-/-) embryos develop, extend neurites, and survive in the absence of neurotrophins, whereas their wild-type counterparts die rapidly unless NGF or BDNF is added to the culture medium. Moreover, NF1 mutant sympathetic neurons survive for extended periods and acquire mature morphology in the presence of NGF-blocking antibodies. The discovery is useful in screening candidate substances for inhibition of neurofibromin action and in therapy for neurodegeneration due to disease or trauma.

Abstract: The present invention relates to nucleic acid sequences encoding intracellular binding proteins. More particularly, the nucleic acid comprises a gene coding for an intracellular single chain antibody specific for a ras oncogene under the control of a promoter, the antibody is functional in mammalian cells, and inhibits the transformation of cells that express a ras oncogene.

Abstract: The invention provides a cells which express a gene or genes, derived from the adenovirus E3 region, which block allograft rejection. One class of genes blocks the intracellular transport and/or intracellular maturation within the cells of proteins called MHC class I products. Without limitation as to theory, it is believed that blocking the appearance of this class of proteins on the transplanted cell's surface, prevents the host's immune system from rejecting the graft. Another class of proteins acts to permit TNF .alpha.-mediated cell cytolysis. In one embodiment, the invention is directed towards engrafting the cells that secrete insulin, which are called alternatively, pancreatic .beta.-cells and islet cells, and thereby provide a treatment of diabetes mellitus.

Abstract: An erythropoietin-inducible, erythroid-specific DNA construct is disclosed. The DNA construct comprises a promoter sequence from a sheep juvenile beta globin gene, an erythroid-specific enhancer sequence from the locus control region of the human beta globin gene and a nucleotide coding sequence of interest.

Abstract: A method for introducing and expressing genes in animal cells is disclosed comprising infecting said animal cells with live invasive bacteria, wherein said bacteria contain a eukaryotic expression cassette encoding said gene. The gene may encode, e.g., a vaccine antigen, an therapeutic agent, an immunoregulatory agent or a anti-sense RNA or a catalytic RNA.

Abstract: A composition comprising genetically altered human neutrophil precursor cells, wherein the cellular component is comprised of at least about 16% human myeloblasts and promyelocytes, which have been derived from neutrophil progenitor cells obtained from peripheral blood, bone marrow or cord blood, and less than about 5% colony forming units (CFU) of at least about 50 cells is provided. An alternative composition comprising genetically altered human neutrophil precursor cells, wherein the cellular component is comprised of at least about 16% CD15+CD11b- cells and less than about 5% colony forming units (CFU) of at least about 50 cells also is provided, wherein at least about 60% of the CD15+CD11b- cells are myeloblasts and promyelocytes.

Abstract: Described herein is an isolated polynucleotide encoding an LPA receptor. Also described is a recombinant DNA molecule comprising a nucleic acid encoding an LPA receptor and expression controlling elements linked therewith, as well as the use of nucleic acid coding for an LPA receptor for expression to obtain a functional receptor protein and for further gene cloning to identify structurally related receptor proteins. Also described herein is LPA receptor as a product of recombinant production in a cellular host. Described is a method of utilizing the LPA receptor in a chemical screening program to identify LPA receptor ligands. The invention further describes antibodies directed to the LPA receptor for use for example in diagnosis of conditions wherein the levels of LPA are altered.

Abstract: Homologous recombination is employed to inactivate genes, particularly genes associated with MHC antigens. Particularly, the .beta..sub.2- microglobulin gene is inactivated for reducing or eliminating the expression of functional Class I MHC antigens. The resulting cells may be used as allogeneic donor cells. Methods for homologous recombination in non-transformed mammalian somatic cells are also described.

Abstract: The chimeric gene is directed by a promoter or fusion of promoters which preferably are regulable and activated by the diabetic process. Preferably, it is obtained by fusion of the human insulin gene to the promoter of PEPCK (P-enolpiruvate carboxiquinasa). Said promoter (fragment -460 bp to +73 bp) is fused to the flank zone 5' of the human insulin gene (-170 bp to +1). The gene of the human insulin contains two coding exons E1 and E2 and two introns A and B. It also relates to an expression vector which allows the expression of insulin in cells which are different from the .beta.-cells of the pancreas, and to a transgenic mouse which expresses said chimeric gene.

Abstract: The present invention features mouse models for Nkx-2.2 gene function and for Nkx-6.1 gene function, wherein the transgenic mouse is characterized by having a defect in Nkx-2.2 gene function or a defect in Nkx-6.1 gene function (where, because Nkx-2.2 acts upstream of Nkx-6.1, a defect in Nkx-2.2 gene function affects Nkx-6.1 gene function) and by having a decreased number of insulin-producing cells relative to a normal mouse. Where the transgenic mouse contains a defect in Nkx-2.2 gene function, the mouse is further characterized by a decreased number of serotonin-producing cells relative to a normal mouse. The transgenic mice may be either homozygous or heterozygous for the Nkx-2.2 or Nkx-6.1 defect.

Abstract: The present invention provides an immune deficient mouse having a human prostate xenograft of locally advanced or metastatic prostate cancer and uses thereof.

Abstract: The invention relates to isolated nucleic acid sequences encoding PRAD1, and fragments thereof. The invention further relates to transgenic mice bearing a transgene which includes a nucleic acid sequence encoding PRAD 1 under transcriptional control of a heterologous promoter, and cells or cell lines derived from such an mice.

Abstract: The use of totipotent embryonic stem cells to provide substantially identical cells for embryo cloning techniques is described. The method includes the culture of loose suspensions of inner cell mass cells of bovine animals to retrieve large populations of stem cells. The invention also describes the use of stem cells in various genetic manipulation techniques.

Abstract: This invention relates to Eimeria proteins with immunogenic properties as well as to DNA sequences encoding these proteins. These proteins can be administered to poultry thereby protecting the birds against coccidiosis. In addition the DNA encoding these proteins can be used for the preparation of a vector vaccine against coccidiosis.

Abstract: A method for specifically suppressing the capacity of a mammal receiving gene therapy to mount an immune response to a given expressed gene of the deficient gene in question, which response is caused by the administration of "foreign" DNA encoding an antigenic protein or vectors comprising such DNA for expression of the deficient gene in the patient receiving gene therapy.

Abstract: The present invention is directed to mice which are genetically altered to be deficient in the normal expression of RXR.gamma., to mice heterozygous for such deficiency, and to cell lines, preferably pluripotent or totipotent cell lines, which are heterozygous or homozygous for such deficiency. The invention further provides mice and cell lines which, in addition to being deficient in RXR.gamma., are genetically altered to be deficient in the expression of RXR.alpha. and/or RXR.beta.. The present invention further provides the use of any of the above mice and cell lines in situations where the absence of RXR.gamma., or the normal expression thereof, is desirable.

Abstract: The present invention discloses nucleic acid molecules encoding WRN gene products, expresion vectors, viral vectors, and host cells suitable for expressing such products.