Abstract: The subject invention concerns novel vectors for the rapid and robust selection for cDNA sequences that encode secreted or membrane-bound proteins. The invention also pertains to methods for cloning secreted or membrane-bound proteins, including proteins encoded by novel members of gene families.

Abstract: This invention relates to the isolation and cloning of the promoter and other control regions of human PPAR? gene. It provides a method for identifying and screening for agents useful for the treatment of diseases and pathological conditions affected by the level of expression of the PPAR? gene. These agents interact directly or indirectly with the promoter or other control regions of the PPAR? gene.

Abstract: The present invention provides for novel chimeric Ad-vectors carrying transgene, or portions of transgenes for stable and efficient gene transfer into diverse cell types or tissues in a CAR- and/or ???3/5-independent manner. Also provided are methods for producing such vectors and the use thereof for gene therapy to target a specific cell type or tissue.

Abstract: The invention includes methods and compositions for the production of high titer recombinant adeno-associated virus (rAAV). The disclosed rAAV are useful in gene therapy applications. Methods are based on the use of recombinant herpes virus vectors and result in highly efficient production of rAAV.

Abstract: The invention hereindescribed relates to a form of cancer therapy which exploits the cytotoxic properties of acetaminophen when converted to NABQI by the metabolic activity of tumour cell specific P450; vectors for use in the delivery of P450 to tumour cells; and therapeutic compositions comprising said vectors.

Abstract: The present invention provides methods of introducing a polynucleotide into a target cell, wherein the method employs a light generating protein coding sequence acting as a reporter. An important advantage of the methods described herein is that drug resistant target cells or target cells having no useful auxotrophic markers can be effectively transformed. The present invention also includes transformed cells produced by the methods described herein. Also described are light generating protein coding sequence modifications, a variety of vectors, and methods of using the transformed cells of the present invention.

Abstract: A method to increase the efficiency of transduction of hematopoietic and other cells by retroviruses includes infecting the cells in the presence of fibronectin or fibronectin fragments. The fibronectin and fibronectin fragments significantly enhance retroviral-mediated gene transfer into the cells, particularly hematopoietic cells including committed progenitors and primitive hematopoietic stem cells. The invention also provides improved methods for somatic gene therapy capitalizing on enhanced gene transfer, hematopoietic cellular populations, and novel constructs for enhancing retroviral-mediated DNA transfer into cells and their use.

Abstract: A method is described for the rapid identification and isolation of cells based on the presence or absence of an ectopically-expressed N-acetyllactosaminide 3-? Galactosyltransferase (?GT) enzyme for the production of ?Galactosyl-(1,3)Galactosyl (?Gal) epitopes on the surface of ?Gal-negative cells. These cells which are genetically modified to express the ?GT enzyme and ?Gal epitopes on glycosylated lipids and proteins of the cell surface are then labeled via an antibody composition which recognizes and binds the ?Gal epitopes on the cell surface. Cells labeled with the anti-?Gal antibody can be isolated by sorting via fluorescence activated cell sorting (FACS), or by magnetic panning techniques. This method is suitable for the rapid positive or negative selection of ?Gal-positive cells from within a population of ?Gal-negative cells without the need to expose cells to antibiotics for any period of time.

Abstract: The present invention is directed to isolated nucleic acid molecules that encode LIM mineralization protein, or LMP. The invention further provides vectors comprising splice variants of nucleotide sequences that encode LMP, as well as host cells comprising those vectors. Moreover, the present invention relates to methods of inducing bone formation by transfecting osteogenic precursor cells with an isolated nucleic acid molecule comprising a nucleotide sequence encoding splice variants of LIM mineralization protein. The transfection may occur ex vivo or in vivo by direct injection of virus or naked plasmid DNA. In a particular embodiment, the invention provides a method of fusing a spine by transfecting osteogenic precursor cells with an isolated nucleic acid molecule having a nucleotide sequence encoding LIM mineralization protein, admixing the transfected osteogenic precursor cells with a matrix and contacting the matrix with the spine.

Abstract: The invention relates to methods and products for deregulating gene transcription in neurodegenerative disease associated with an expanded polyglutamine tract in mutant proteins. The invention is useful for preventing and treating diseases associated with expanded polyglutamine tracts, including Huntington's disease. The methods and compositions of the invention are also useful for identifying additional pharmaceutical agents for preventing and treating diseases associated with expanded polyglutamine tracts.

Abstract: The invention refers to the production of recombinant gene products from cultures of the yeast Zygosaccharomyces bailii strains transformed with expression vectors bearing the gene coding for said proteins.

Abstract: The present invention provides polypeptides comprising an immunogenic epitope of a M. vaccae protein, polynucleotides encoding such polypeptides, and fusion proteins comprising at least one such polypeptide, together with genetic constructs comprising at least one inventive polynucleotide. Compositions comprising such polypeptides, polynucleotides, fusion proteins and/or genetic constructs may be employed in the treatment of infectious diseases and immune disorders.

Abstract: The present invention relates to methods of inducing neuronal production in the brain, recruiting neurons to the brain, and treating a neurodegenerative condition by providing a nucleic acid construct encoding a neurotrophic factor, and injecting the nucleic acid construct intraventricularly into a subject's brain.

Abstract: Disclosed is a developmental animal model of temporal lobe epilepsy and other seizure-related disorders. In particular, the invention provides a method of inducing a permanent change in the neurological development of a rodent, such as a rat, comprising daily administration of low doses of a kainate receptor agonist to the animal in the second postnatal week, wherein after treatment with the kainate receptor agonist the animal exhibits reproducible seizure-like symptoms when exposed to mild to moderate stressors. Rats treated using the above method are particularly useful as a non-human system for studying temporal lobe epilepsy, as well as for studying the efficacy of potential anti-epileptic compounds and pharmaceutical preparations.

Abstract: The present invention is directed towards a method of regulating smooth muscle tone, comprising the introduction, into smooth muscle cells of a subject, of a DNA sequence encoding a protein involved in the regulation of smooth muscle tone, and expression of the DNA sequence in a sufficient number of smooth muscle cells of the subject to regulate smooth muscle tone in the subject. Specifically, the invention provides methods of gene therapy for treating erectile dysfunction, bladder dysfunction, and other smooth muscle disorders. The present invention also provides recombinant viral and non-viral vectors comprising DNA encoding a protein involved in the regulation of smooth muscle tone. Further provided by the present invention is a smooth muscle cell which expresses a gene encoding a protein involved in the regulation of smooth muscle tone.

Abstract: The present invention relates to a composition and method for delivery of biological material, especially nucleic acids into target cells and into the nucleus.

Abstract: The present invention provides novel expression vectors which permit tight regulation of gene expression in eucaryotic cells. More specifically, the invention provides DNA vectors comprising nucleotide sequences that are transcribed to form RNA molecules which are then replicated by a temperature-sensitive replicase to form additional RNA molecules. The RNA molecules produced by replication contain a nucleotide sequence which may be translated to produce a protein of interest or which encode one or more untranslated RNA molecules. Also provided are methods for producing heterologous proteins and untranslated RNA molecules. Further provided are methods for administering heterologous proteins and untranslated RNA molecules to individuals. In addition, pharmaceutical compositions are provided comprising the DNA and RNA molecules of the invention and a pharmaceutically acceptable carrier.

Abstract: A method for preventing, delaying the onset of or treating diabetes in a patient comprising selecting a patient who is susceptible to developing diabetes, who is developing diabetes or who is diabetic and administering to the patient one or more than one dose of a pharmaceutical agent comprising a polynucleotide encoding a secreted exogenous protein, such as a secreted luciferase or a secreted form of human glutamic acid decarboxylase.

Abstract: Dominant negative alleles of human mismatch repair genes can be used to generate hypermutable cells and organisms. By introducing these genes into cells and transgenic animals, new cell lines and animal varieties with novel and useful properties can be prepared more efficiently than by relying on the natural rate of mutation. Methods of generating mutations in genes of interest and of making various cells mismatch repair defective through the use of chemicals to block mismatch repair in in vivo are disclosed.

Abstract: The invention relates to novel degradation resistant FGF-1, and methods for producing and using the same. More specifically, the invention relates to identification of a thrombin degradation resistant FGF-1, an a nucleic acid encoding the same. The thrombin degradation resistant FGF-1 can elicit responses that are otherwise typically impeded by degradation of FGF-1 by thrombin. Thrombin degradation resistant FGF-1 is an important molecule for effecting an FGF-1 response that would be otherwise inhibited by thrombin. Thus, the present invention provides a powerful therapeutic for diseases or disorders wherein an FGF-1 response can mediate a reduction in the frequency or intensity of a symptom of the disease or disorder but for degradation of FGF-1 before it can effect the response.