Abstract: The invention relates to an isolated nucleic acid encoding a eukaryotic Survival of Motor Neuron-Interacting Protein 1 (SIP1), compositions comprising SIP1 and SIP1 and the spinal muscular atrophy (SMA) disease gene product Survival of Motor Neuron protein (SMN), and diagnostic and therapeutic assays directed to SMA. The invention also relates to another protein that specifically interacts with SMN and is a component of gems, designated Gemin3, and the nucleic acid encoding the protein. Additionally, the invention relates to a novel cell line wherein the endogenous SMN genes have been deleted and where an exogenous nucleic acid encoding SMN has been inserted into the cell such that expression of SMN in the cell is under the control of an inducible promoter. This novel cell line provides a stable genetic system for the study of SMA and for the development of SMA therapeutics.

Abstract: This invention provides methods of modifying feeding behavior, including increasing or decreasing food consumption, e.g., in connection with treating obesity, bulimia or anorexia. These methods involve administration of compounds that are selective agonists or antagonists for the Y5 receptor. One such compound has the structure: In addition, this invention provides an isolated nucleic acid molecule encoding a Y5 receptor, an isolated Y5 receptor protein, vectors comprising an isolated nucleic acid molecule encoding a Y5 receptor, cells comprising such vectors, antibodies directed to the Y5 receptor, nucleic acid probes useful for detecting nucleic acid encoding Y5 receptors, antisense oligonucleotides complementary to any unique sequences of a nucleic acid molecule which encodes a Y5 receptor, and nonhuman transgenic animals which express DNA encoding a normal or a mutant Y5 receptor.

Abstract: The invention provides a novel method for down-regulating the biological activity of osteoprotegerin ligand (OPGL, TRANCE) thereby rendering possible the treatment/amelioration of diseases characterized by excessive loss of bone mass, e.g. osteoporosis. Down-regulation is effected by inducing an immune response against OPGL in an individual in need thereof. Immune responses can be raised by classical immunization with immunogenic variants of OPGL or by nucleic acid immunization where the nucleic acids encode the OPGL variant. The invention also pertains to compositions, polypeptides and nucleic acids useful in the invention, as well as to vectors and transformed host cells useful in the preparation thereof.

Abstract: FK506 and geldanamycin promote nerve regeneration by a common mechanism that involves the binding of these compounds to polypeptide components of steroid receptor complexes other than the steroid hormone binding portion of the complex (FKBPS52 and hsp90, respectively). These and other agents cause hsp90 dissociation from steroid receptor complexes or block association of hsp90 with steroid receptor complexes.

Abstract: The invention relates to methods for identifying candidate pharmacological agents to be used in the treatment and/or prevention of Alzheimer's disease and/or related pathological conditions.

Abstract: The present invention relates to a novel human K+ ion channel, to nucleic acid molecules encoding the same and to vectors comprising said nucleic acid molecules. The invention additionally relates to antibodies specifically directed to the novel K+ ion channel and to pharmaceutical compositions and diagnostic kits containing at least one of the above-mentioned components. Furthermore, the present invention relates to methods of treating a disease caused by malfunction of the polypeptide of the present invention or by the (over)expression of the nucleic acid molecule of the invention comprising administering an inhibitor of said (over)expression or of ion channel function or an inhibitor abolishing said malfunction to a patient in need thereof. Methods of devising drugs for treating or preventing the above-mentioned disease, methods of inhibiting cell proliferation and methods of prognosing cancer are additional embodiments comprised by the present invention.

Abstract: Human polypeptides and DNA (RNA) encoding such polypeptides and a procedure for producing such polypeptides by recombinant techniques is disclosed. Also disclosed are methods for utilizing such polypeptides for therapeutic purposes. Antagonists against such polypeptides and their use as a therapeutic are also disclosed. Also disclosed are diagnostic methods for detecting disease which utilize the sequences and polypeptides.

Abstract: Beta-amyloid peptide (&bgr;A) is a major fibrillar component of neuritic plaques in Alzheimer's disease (AD) brains and is related to the pathogenesis of the disease. The present invention provides a method using Poly-L-Lysine to dissolve preformed &bgr;A fibrils in vitro. Its efficiency is instantaneous. Poly-L-Lysine offers the simplest and most effective way to dissolve preformed &bgr;A fibrils. The method of this present invention can be used as a universal dissolver of all types of oligomeric &bgr;-sheet conformation, precursor of the fibrils. Poly-L-Lysine may also be useful as a future universal therapeutic agent to prevent and/or retard amyloidogenesis in vivo AD and other types of amyloid related disorders.

Abstract: Disclosed are (1) a human luteinizing hormone-human chorionic gonadotropin receptor protein, (2) a DNA comprising a CDNA segment coding for a human luteinizing hormone-human chorionic gonadotropin receptor protein, SEQ ID NO:2 (3) a transformant carrying a DNA comprising a cDNA segment SEQ ID NO:1 coding for a human luteinizing hormone-human chorionic gonadotropin receptor protein, and (4) a method for preparing a human luteinizing hormone-human chorionic gonadotropin receptor protein which comprises cultivating the transformant described in (3), accumulating a protein SEQ ID NO:2 in a culture broth, and collecting the same, whereby the structure and properties of the receptor protein are made clear and the mass production thereof by recombinant technology is pioneered.

Abstract: The present invention relates to a novel Neutrokine-alpha, and a splice variant thereof designated Neutrokine-alphaSV, polynucleotides and polypeptides which are members of the TNF family. In particular, isolated nucleic acid molecules are provided encoding the human Neutrokine-alpha and/or Neutrokine-alphaSV polypeptides, including soluble forms of the extracellular domain. Neutrokine-alpha and/or Neutrokine-alphaSV polypeptides are also provided as are vectors, host cells and recombinant methods for producing the same. The invention further relates to screening methods for identifying agonists and antagonists of Neutrokine-alpha and/or Neutrokine-alphaSV activity. Also provided are diagnostic methods for detecting immune system-related disorders and therapeutic methods for treating immune system-related disorders.

Abstract: Growth differentiation factor, Lefty-2, is disclosed along with its polynucleotide sequence and amino acid sequence. Also disclosed are diagnostic and therapeutic methods of using the Lefty-2 polypeptide and polynucleotide sequences.

Abstract: The present invention can be used in pharmacology specifically in the preparation of interferon-containing compositions, which are capable of conserving their biological activity and can be administrated intranasally, e.g. in the preparation of nasal drops. This invention essentially refers to an antiviral agent in the form of nasal drops that contains a genetically engineered alpha, beta or gamma interferon with a viscosity of (1.1-30.0)* 10 Pascal ·second, a biocompatible polymer and a buffer mixture. The agent may further include an antioxidant, and the ingredients are contained in the following amounts per ml buffer mixture: 1,000 to 5,000 IU of genetically engineered interferon; 0.005 to 0.714 g of biocompatible polymer; and 0.0001 to 0.0008 g of an antioxidant. TRILON B® (disodium salt of EDTA) is used as the antioxidant, whereas polyvinylpyrrolidone and/or polyethylene oxide is (are) used as the biocompatible polymer(s) at polyvinylpyrrolidone/polyethylene oxide ratio of 1:1-50.

Abstract: The present invention is directed to novel polypeptides and to nucleic acid molecules encoding those polypeptides. Also provided herein are vectors and host cells comprising those nucleic acid sequences, chimeric polypeptide molecules comprising the polypeptides of the present invention fused to heterologous polypeptide sequences, antibodies which bind to the polypeptides of the present invention and to methods for producing the polypeptides of the present invention.

Abstract: The invention features a method of identifying, evaluating or making a compound or agent, e.g., a candidate compound or agent, for treatment of a disorder characterized by insulin resistance. The method includes evaluating the ability of a compound or agent to bind IKK-&bgr; or modulate IKK-&bgr; activity, to thereby identify a compound or agent for the treatment of a disorder characterized by insulin resistance. The invention also features compounds for treating insulin resistance identified by such methods, and methods of treating a subject having a disorder characterized by insulin resistance by administering such agents.

Abstract: The subject matter of the invention is directed to a single-chain insulin analog that is used to treat diabetes by gene therapy methods.

Abstract: A method for treating a patient that has inadequate intestinal function is described. Administering leptin to a subject increases the intestinal function beyond that for a normal intestine and beyond that of a normal adaptive response. Further, administering leptin to a subject results in an increase in amino acid absorption, sugar absorption, mucosal mass, transport mechanisms for amino acids, or transport mechanisms for sugars. The method may be used for treating subjects have conditions such as short bowel syndrome, inflammation of the bowel, necrotizing enterocolitis, intestinal atresia, midgut volvulus, severe acute gastroenteritis, chronic gastroenteritis, cholera, chronic infections of the bowel, immunologic disorders affecting the small intestine, and inflammatory bowel disease such as, chronic ulcerative colitis and Crohn's Disease.

Abstract: Human Ect2 polypeptide, fragments and derivatives, along with vectors and host cells for expression and production of Ect2 polypeptide are provided. Various methods of screening for agents that modulate interaction of Ect2 with an Ect2 binding agent, including high throughput methods, are also provided.

Abstract: The invention relates to novel granulopoietic activity (GPA) proteins and nucleic acids. The invention further relates to the use of the GPA proteins in the treatment of G-CSF related disorders.

Abstract: Purified BMP-16 proteins and processes for producing them are disclosed. DNA molecules encoding the BMP-16 proteins are also disclosed. The proteins may be used in the treatment of bone, cartilage, other connective tissue defects and disorders, including tendon, ligament and meniscus, in wound healing and related tissue repair, as well as for treatment of disorders and defects to tissues which include epidermis, nerve, muscle, including cardiac muscle, and other tissues and wounds, and organs such as liver, lung, cardiac, pancreas and kidney tissue. The proteins may also be useful for the induction of growth and/or differentiation of undifferentiated embryonic and stem cells.

Abstract: The present invention provides a method for ameliorating &bgr;-globin disorders in a mammal. In one aspect of the invention, the treatment involves ex vivo treatment of early erythroid progenitor cells that leads to an increase in the relative amounts of cells subsequently expressing and accumulating HbF. The cell treatment is to be followed by transplantation of the modified cells. In another aspect of the invention, the same modification of progenitor cells occurs in vivo. Both treatments are based on the novel discovery that the modification can be performed very early in the erythroid maturation process, without disturbance of the subsequent proliferation and maturation of the erythroid precursor. The present invention also provides a procedure for the monitoring of &bgr;-globinopathies and the response of a patient to treatment.