Abstract: The invention features antisense oligonucleotides and methods of using these antisense oligonucleotides for inhibiting HCV RNA translation.

Abstract: Transgenic cell and animal models for Alzheimer's disease are described. Cells of the animals and the cell models themselves comprise a recombinant DNA construct comprising a control sequence and, under the control of the control sequence, a DNA sequence encoding a kinase that is capable, directly or indirectly, of modulating the phosphorylation of the microtubule-forming protein tau. The transgenic cells and animals may be used for testing potential therapeutic agents for Alzheimer's disease.

Abstract: A method of causing production and secretion into the bloodstream of a human patient of a biologically active enzyme for which the human patient suffers a deficiency; the method involves introducing into the human patient donor bone marrow stromal cells which have been transfected with a gene encoding the enzyme, so that the introduced cells can adhere to a bone cavity surface of the patient and produce and secrete the active enzyme.

Abstract: Embryonic stem cells derived from an inbred mouse strain selected from the group consisting of C3H/HeN, C57BL/6N, DBA/1J, and BALB/c strains, for example, C-2 cell having the accession number FERM BP-5933 and C-6 cell having the accession number FERM BP-5934 are disclosed. The embryonic stem cells of the present invention are derived from genetically complete inbred strains, and therefore, they are extremely useful for close genetic research.

Abstract: The present invention relates to a method of examining the neurovirulence of a polio virus, which comprises inoculating a polio virus into the spinal cord of a transgenic mouse comprising a gene for a polio virus receptor.

Abstract: A combination of retroviral and adenoviral vectors are used for high efficiency gene transfer into hepatocytes, resulting in long term gene expression. Hepatocytes are transduced in vivo with a recombinant adenovirus vector that expresses a molecule capable of inducing hepatocyte regeneration, such as urokinase plasminogen activator (uPA) or tissue plasminogen activator (tPA), resulting in a high rate of liver regeneration. During the regenerative phase, ex vivo or in vivo retroviral-mediated gene transfer into hepatocytes results in greater transduction efficiencies. The compositions and methods thus provide new means for gene therapy, and transgenic non-human animals useful in developing new therapeutic and preventative agents.

Abstract: The present invention provides a transgenic mouse comprising a disrupted hepsin gene. In particular, the invention provides methods of making the transgenic mouse comprising the disrupted hepsin gene by utilizing a hepsin targeting vector for homologous recombination in mouse embryonic stem cells. Also, nucleotide and amino acid hepsin sequences are disclosed.

Abstract: The invention is directed to a transgene expression system comprising a transcription unit which contains a transgene operably linked to expression control sequences, preferably the CMV promoter, and which is delivered simultaneously with all or part of the adenovirus E4 genomic region to a cell in order to facilitate persistent expression of the transgene. The components of the transgene expression system can be delivered by vectors including plasmids and/or viruses and may be complexed with cationic amphiphiles to facilitate entry into a cell. The invention is also directed to methods for the production of the transgene expression system. The invention is further directed to compositions that contain the transgene expression system and to methods for the use of such compositions to deliver transgenes encoding biologically active proteins to cells.

Abstract: A method for preventing the formation of platelet-rich clots or thrombus, and for deaggregating platelet-rich clots or thrombus, using hementin, a polypeptide derived from leech salivary glands. Hementin may be administered for this purpose in a pharmaceutically acceptable carrier or excipient, with or without additional anticoagulants such as hirudin, hirudin analogues, or an inhibitor of factor Xa.

Abstract: Methods for increasing the nephron mass of a mammalian recipient are disclosed. A metanephros from an allogenic or xenogeneic mammalian donor is implanted next to a recipient's omentum or under the renal capsule of the recipient's kidney. The metanephros becomes vascularized by the recipient's blood vessels, forming a chimeric kidney that produces urine and develops a ureter that facilitates externalization of the urine. A ureter to ureter anastomosis can be subsequently performed to provide fluid communication between the chimeric kidney ureter and a ureter of the recipient.

Abstract: A gene delivery system is made of enzymatically degradable polymeric cation and nucleic acid (DNA or RNA) nanospheres optionally with a linking moiety or a targeting ligand attached to the surface. The delivery system can be made by a simple method of coacervation. Targeting ligands can be attached to the nanosphere directly or via a linking moiety. The linkage design allows the attachment of any molecule onto the nanosphere surface including antibodies, cell adhesion molecules, hormones and other cell-specific ligands.

Abstract: The invention provides novel mutant S182 sequences, methods of diagnosing Alzheimer's disease using these novel mutant S182 genes, a model system for Alzheimer's disease comprising a mutant S182 gene, and methods of identifying mutations in genes homologous to the S182 gene.

Abstract: Nucleic acids are compacted, substantially without aggregation, to facilitate their uptake by target cells of an organism to which the compacted material is administered. The nucleic acids may achieve a clinical effect as a result of gene expression, hybridization to endogenous nucleic acids whose expression is undesired, or site-specific integration so that a target gene is replaced, modified or deleted. The targeting may be enhanced by means of a target cell-binding moiety. The nucleic acid is preferably compacted to a condensed state.

Abstract: A method for introducing foreign nucleic acid sequences into marine mollusks. A pantropic retroviral vector containing a foreign gene sequence is introduced into fertilized mollusk embryos by electroporation. The gene sequence becomes integrated into the host DNA and encodes a functional protein product. This method has implications in the introduction of disease-resistance and growth-accelerating genes into mollusks.

Abstract: Nucleic and amino acid sequences for a novel cell surface transmembrane glycoprotein designated ClqR.sub.P are taught. A method by which the nucleic acid sequence encoding ClqR.sub.P may be used to modulate the role of the immune system is also described. Transgenic animals created with heterologous DNA sequence encoding ClqR.sub.P are described as well as antibodies directed against the ClqR.sub.P protein. A method of hybridization for ClqR.sub.P nucleic acid using oligonucleotide probes based on the ClqR.sub.P nucleic acid sequence and methods for detecting a hybrid probe:target ClqR.sub.P duplex are also taught including a kit for such detection.

Abstract: Inhibition of eucaryotic pathogens and neoplasms and stimulation of lymphocytes and fibroblasts with lytic peptides such as cecropins and sarcotoxins. Eucaryotic cells are contacted with cecropin or sarcotoxin, or a synergistic combination of cecropins or sarcotoxin with lysozyme, in an amount effective to lyse or inhibit the cells. Target cells include eucaryotic microorganisms such as protozoa, e.g. T. cruzi and P. falciparum, mammalian lymphomas and leukemias, and cells infected with intracellular pathogens such as viruses, bacteria and protozoa. Also disclosed is a method for stimulating proliferation of lymphocytes and fibroblasts by contacting such cells with an effective amount of cecropin or sarcotoxin. The methods may be in vitro or in vivo.

Abstract: Disclosed are genes encoding novel CF monomer proteins which have cystic fibrosis transmembrane conductance regulator (CFTR) protein function.

Abstract: A method for bacterially producing IGF-I is disclosed in which Gram-negative bacteria are caused to express a gene consisting of a lamB or ompF signal sequence operatively joined to a DNA sequence encoding IGF-I and producing IGF-I which is secreted into the periplasmic space of the bacteria.

Abstract: Disclosed is a method of localized immunosuppression which may be used for preventing graft rejection or for preventing tissue destruction due to autoimmune disease. Also disclosed is a protein suppressor factor that is secreted by cloned anergic T-cells, blocks interleukin 2 (IL-2) stimulated T-cell proliferation, has an apparent molecular weight of between 10 and 30 kilodaltons, can be inactivated by heating to 65.degree. C. for 15 minute, blocks interleukin 4 (IL-4) stimulated T-cell proliferation in vitro, is non-cytotoxic to T-cells, and does not inhibit the production of IL-2 by T-cells in vitro.

Abstract: A method and means for protecting cells and transplanted organs from the effects of activated complement proteins generated in blood serum or plasma by introducing the gene for CD59 into the cells to be protected is described. In an example of the method, protection against the pore-forming activity of the human C5b-9 proteins was conferred on CHO cells by transfection with cDNA encoding the human complement regulatory protein CD59.