MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY (USA)
Inventor
Steinmetz, Lars
Kornienko, Julia
Fenzl, Kai
Rodríguez-Martínez, Marta
Gotthardt, Michael
Abstract
The present invention relates to agents that increase the amount of a protein comprising a mutated RS domain amino acid sequence in the nucleus of a cell and/or decreases the amount of the same protein in the cytoplasm of said cell, for use in the prevention or treatment of a disease or condition that is related to the cytoplasmic mislocalization and/or the formation of granules and/or an aberrant splicing activity of the protein comprising the mutated RS domain amino acid sequence. In particular, the protein comprising a mutated RS domain amino acid sequence is RBM20 or an ortholog thereof, and/or the nuclear transporter protein is transportin 3 (TNPO3) or an ortholog thereof. Preferably, the disease or condition is myopathy, in particular dilated cardiomyopathy (DCM). The present invention further relates to a method for identifying suitable agents used to increase the amount of a protein comprising a mutated RS domain amino acid sequence in the nucleus of a cell and/or to decrease the amount of the same protein in the cytoplasm of said cell.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
2.
INCREASE OF PROTEIN EXPRESSION AND SECRETION BY ARTIFICIAL CO-EXPRESSION OF HDLBP/VIGILIN
MAX DELBRUCK-CENTRUM FUR MOLEKULARE MEDIZIN (Germany)
HUMBOLDT-UNIVERSITAT ZU BERLIN (Germany)
Inventor
Landthaler, Markus
Zinnall, Ulrike
Minia, Igor
Abstract
The invention is related to a method for producing a secreted recombinant protein of interest in a cell, wherein the method comprises artificially co-expressing a protein different from the secreted recombinant protein of interest.
C12N 5/10 - Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
HUMBOLDT-UNIVERSITÄT ZU BERLIN (Germany)
Inventor
Landthaler, Markus
Zinnall, Ulrike
Minia, Igor
Abstract
The invention is related to a method for producing a secreted recombinant protein of interest in a cell, wherein the method comprises artificially co-expressing a protein different from the secreted recombinant protein of interest.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
C12N 15/67 - General methods for enhancing the expression
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
4.
METHOD FOR GENE REPAIR IN PRIMARY HUMAN MUSCLE STEM CELLS (SATELLITE CELLS) IN VITRO AND GENETICALLY REPAIRED HUMAN MUSCLE STEM CELL
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Spuler, Simone
Escobar, Helena
Abstract
The invention relates to a method for gene repair in primary human muscle stem cells (satellite cells) in vitro comprising the following steps: providing a sample of an isolated muscle-fiber containing tissue sample collected from at least one patient with a monogenic muscle disease, wherein the monogenic muscle disease is caused by at least one mutation in at least one gene encoding for at least one muscle protein; isolating and cultivating primary stem cells from said muscle-fiber containing tissue sample, and correcting the at least one mutation in the at least one gene encoding for at least one muscle protein in the cultivated primary stem cells by targeted modification of the at least one mutation by gene editing using CRISPR/Cas-based tools. The invention relates also to genetically repaired human muscle stem cell obtained in such a method.
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression
C07K 14/435 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
C07K 14/78 - Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
5.
Analogs of CYP-Eicosanoids for Use in Treating or Preventing a Disorder Associated with Neovascularization and/or Inflammation
Max Delbrück-Centrum für Molekulare Medizin (Germany)
Inventor
Fischer, Robert
Schunck, Wolf-Hagen
Müller, Dominik
Wesser, Tim
Konkel, Anne
Lossie, Janine
Abstract
The present invention relates to compounds according to general formula (I) which are metabolically robust analogues of bioactive lipid mediators derived from omega-3 polyunsaturated fatty acids (n-3 PUFAs) for use in treating or reducing the risk of developing or preventing: (i) neovascularization and/or (ii) inflammatory disorder, in particular, ophthalmic disorders associated with neovascularization and/or inflammation.
A61K 31/165 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide
A61K 31/192 - Carboxylic acids, e.g. valproic acid having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid
A61K 31/22 - Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin
A61K 31/341 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide not condensed with another ring, e.g. ranitidine, furosemide, bufetolol, muscarine
A61K 31/351 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom not condensed with another ring
A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
A61K 31/40 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
6.
Metabolically robust analogs of CYP-eicosanoids for the treatment of cardiac disease
Max Delbruck-Centrum Fur Molekulare Medizin (Germany)
The Board of Regents of the University of Texas System (USA)
Omeicos Therapeutics GmbH (Germany)
Inventor
Fischer, Robert
Konkel, Anne
Wesser, Tim
Westphal, Philipp
Schunk, Wolf-Hagen
Westphal, Christina
Falck, John Russell
Abstract
The present invention relates to compounds according to general formula (I) which are metabolically robust analogues of bioactive lipid mediators derived from omega-3 polyunsaturated fatty acids (n-3 PUFAs). The present invention further relates to compositions containing one or more of these compounds and to the use of these compounds or compositions for the treatment or prevention of cardiovascular diseases.
A61P 9/00 - Drugs for disorders of the cardiovascular system
A61K 31/08 - Ethers or acetals acyclic, e.g. paraformaldehyde
A61K 31/202 - Carboxylic acids, e.g. valproic acid having a carboxyl group bound to an acyclic chain of seven or more carbon atoms, e.g. stearic, palmitic or arachidic acid having three or more double bonds, e.g. linolenic acid
C07C 233/56 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having carbon atoms of carboxamide groups bound to carbon atoms of carboxyl groups, e.g. oxamides
C07C 275/20 - Derivatives of urea, i.e. compounds containing any of the groups the nitrogen atoms not being part of nitro or nitroso groups having nitrogen atoms of urea groups bound to acyclic carbon atoms of an unsaturated carbon skeleton
C07C 311/17 - Sulfonamides having sulfur atoms of sulfonamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the sulfonamide groups bound to hydrogen atoms or to an acyclic carbon atom to an acyclic carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
A61P 9/10 - Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
FORSCHUNGSVERBUND BERLIN E.V. (Germany)
Inventor
Bader, Michael
Specker, Edgar
Matthes, Susann
Schütz, Anja
Mallow, Keven
Grohmann, Maik
Nazaré, Marc
Abstract
The invention relates to a xanthine derivative defined by chemical formula I or a salt thereof, its use as a medicament, especially for use in the treatment of serotonin-related diseases or disorders, and a pharmaceutical preparation comprising the xanthine derivative. The novel xanthine compounds are capable of inhibiting tryptophan hydroxylases (TPH) involved in the biosynthesis of serotonin and are effective in influencing the serotonin level in the body.
C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or
8.
ANALOGS OF CYP-EICOSANOIDS FOR USE IN TREATING OR PREVENTING A DISORDER ASSOCIATED WITH NEOVASCULARIZATION AND/OR INFLAMMATION
MAX DELBRUCK-CENTRUM FUR MOLEKULARE MEDIZIN (Germany)
Inventor
Fischer, Robert
Schunck, Wolf-Hagen
Muller, Dominik
Wesser, Tim
Konkel, Anne
Lossie, Janine
Abstract
The present invention relates to compounds according to general formula (I) which are metabolically robust analogues of bioactive lipid mediators derived from omega-3 polyunsaturated fatty acids (n-3 PUFAs) for use in treating or reducing the risk of developing or preventing: (i) neovascularization and/or (ii) inflammatory disorder, in particular, ophthalmic disorders associated with neovascularization and/or inflammation.
A61K 31/192 - Carboxylic acids, e.g. valproic acid having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid
A61K 31/22 - Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin
A61K 31/341 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide not condensed with another ring, e.g. ranitidine, furosemide, bufetolol, muscarine
A61K 31/351 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom not condensed with another ring
A61K 31/40 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
A61K 31/41 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which is nitrogen, e.g. tetrazole
The invention relates to a system and method for introducing DNA into cells. In particular, the invention relates to a method for introducing single or multiple copies of a DNA sequence or gene of interest into a cell comprising providing: a) a "copy and paste" transposase; and b) a construct comprising a DNA sequence or gene of interest flanked by a "copy and paste" transposon terminal sequence, such as an LTS or RTS. A novel "copy and paste" transposon of the Helitron family is described along with systems for using the corresponding transposase in methods for introducing DNA into cells, for example, to generate cell lines for use in protein production, cell and gene therapy or as reference standards.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
GENETIC INFORMATION RESEARCH INSTITUTE (USA)
Inventor
Buerckstuemmer, Tilmann
Kapitonov, Vladimir Vyacheslavovich
Grabundzija, Ivana
Ivics, Zoltan
Abstract
The invention relates to a system and method for introducing DNA into cells. In particular, the invention relates to a method for introducing single or multiple copies of a DNA sequence or gene of interest into a cell comprising providing: a) a "copy and paste" transposase; and b) a construct comprising a DNA sequence or gene of interest flanked by a "copy and paste" transposon terminal sequence, such as an LTS or RTS. A novel "copy and paste" transposon of the Helitron family is described along with systems for using the corresponding transposase in methods for introducing DNA into cells, for example, to generate cell lines for use in protein production, cell and gene therapy or as reference standards.
BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM (USA)
MAX DELBRUCK-CENTRUM FUR MOLEKULARE MEDIZIN (Germany)
Inventor
Fischer, Robert
Konkel, Anne
Wesser, Tim
Westphal, Philipp
Schunk, Wolf-Hagen
Westphal, Christina
Falck, John Russel
Abstract
The present invention relates to compounds according to general formula (I) which are metabolically robust analogues of bioactive lipid mediators derived from omega-3 polyunsaturated fatty acids (n-3 PUFAs).The present invention further relates to compositions containing one or more of these compounds and to the use of these compounds or compositions for the treatment or prevention of cardiovascular diseases.
C07C 275/20 - Derivatives of urea, i.e. compounds containing any of the groups the nitrogen atoms not being part of nitro or nitroso groups having nitrogen atoms of urea groups bound to acyclic carbon atoms of an unsaturated carbon skeleton
A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
A61P 9/00 - Drugs for disorders of the cardiovascular system
C07C 233/56 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having carbon atoms of carboxamide groups bound to carbon atoms of carboxyl groups, e.g. oxamides
C07C 311/17 - Sulfonamides having sulfur atoms of sulfonamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the sulfonamide groups bound to hydrogen atoms or to an acyclic carbon atom to an acyclic carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM (USA)
MAX DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Fischer, Robert
Konkel, Anne
Wesser, Tim
Westphal, Philipp
Schunk, Wolf-Hagen
Westphal, Christina
Falck, John Russel
Abstract
The present invention relates to compounds according to general formula (I) which are metabolically robust analogues of bioactive lipid mediators derived from omega-3 polyunsaturated fatty acids (n-3 PUFAs).The present invention further relates to compositions containing one or more of these compounds and to the use of these compounds or compositions for the treatment or prevention of cardiovascular diseases.
C07C 275/20 - Derivatives of urea, i.e. compounds containing any of the groups the nitrogen atoms not being part of nitro or nitroso groups having nitrogen atoms of urea groups bound to acyclic carbon atoms of an unsaturated carbon skeleton
C07D 309/14 - Nitrogen atoms not forming part of a nitro radical
C07C 311/17 - Sulfonamides having sulfur atoms of sulfonamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the sulfonamide groups bound to hydrogen atoms or to an acyclic carbon atom to an acyclic carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
A61P 9/00 - Drugs for disorders of the cardiovascular system
C07C 233/56 - Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having carbon atoms of carboxamide groups bound to carbon atoms of carboxyl groups, e.g. oxamides
C07D 263/48 - Nitrogen atoms not forming part of a nitro radical
C07D 295/192 - Radicals derived from carboxylic acids from aromatic carboxylic acids
C07D 307/68 - Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen
A61K 31/17 - Amides, e.g. hydroxamic acids having the group N—C(O)—N or N—C(S)—N, e.g. urea, thiourea, carmustine
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
FORSCHUNGSVERBUND BERLIN E.V. (Germany)
Inventor
Bader, Michael
Specker, Edgar
Matthes, Susann
Schütz, Anja
Mallow, Keven
Grohmann, Maik
Nazaré, Marc
Abstract
The invention relates to a xanthine derivative defined by chemical formula I or a salt thereof, its use as a medicament, especially for use in the treatment of serotonin-related diseases or disorders, and a pharmaceutical preparation comprising the xanthine derivative. The novel xanthine compounds are capable of inhibiting tryptophan hydroxylases (TPH) involved in the biosynthesis of serotonin and are effective in influencing the serotonin level in the body.
C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or
A61K 31/522 - Purines, e.g. adenine having oxo groups directly attached to the heterocyclic ring, e.g. hypoxanthine, guanine, acyclovir
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
NATIONAL UNIVERSITY OF SINGAPORE (Singapore)
Inventor
Schäfer, Sebastian
Huebner, Norbert
Cook, Stuart, A.
Abstract
The application relates to a method for diagnosing dilated cardiomyopathy (DCM) in a subject or for assessing the risk of a subject to acquire DCM or the risk of heart failure comprising: (i) determining in a sample of a subject to be diagnosed the sequence of one or more polymorphisms in a (first) panel of genes, wherein the panel of genes comprises the genes of the group consisting of PIEZO1, PLEC, HELZ2, NACAD, PKD1, IGSF10, TNRC18, UNC13B, VWF, and XIRP2; (ii) comparing the determined sequence to the sequence of said polymorphism in a control derived from a control subject (control sequence), the control subject not suffering from dilated cardiomyopathy; and (iii) attributing the presence of dilated cardiomyopathy or the risk of acquiring DCM or the risk of heart failure to said subject to be diagnosed if one or more determined sequences differ from the respective sequence in said control sequence. Furthermore, the application relates to a kit or array for diagnosing dilated cardiomyopathy (DCM) in a subject or for assessing the risk of a subject to acquire DCM comprising means for determining the sequence of one or more polymorphisms in a (first) panel of genes comprising probes for detecting a one or more SNP within each gene of the first panel of genes.
MAX-DELRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Lipp, Martin
Oden, Felix
Höpken, Uta
Müller, Gerd
Daumke, Oliver
Marino, Stephen
Olal, Daniel
Abstract
The invention relates to antibodies or antibody fragments that bind CD269 (BCMA), thereby disrupting the interaction between CD269 and its native ligands (BAFF and APRIL), and their use in the treatment of plasma cell-mediated diseases such as multiple myeloma and autoimmune diseases.
C07K 16/30 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
A61K 39/00 - Medicinal preparations containing antigens or antibodies
The present invention relates to compounds according to general formula (I) which are analogues of epoxymetabolites produced by cytochrome P450 (CYP) enzymes from omega-3 (n-3) polyunsaturated fatty acids (PUFAs). The present invention further relates to compositions containing one or more of these compounds and to the use of these compounds or compositions for the treatment or prevention of conditions and diseases associated with inflammation, proliferation, hypertension, coagulation, immune function, pathologic angiogenesis, heart failure and cardiac arrhythmias.
C07C 309/21 - Sulfonic acids having sulfo groups bound to acyclic carbon atoms of an acyclic unsaturated carbon skeleton containing nitrogen atoms, not being part of nitro or nitroso groups, bound to the carbon skeleton
A61K 31/165 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide
A61K 31/185 - AcidsAnhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
A61K 31/41 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which is nitrogen, e.g. tetrazole
A61K 31/661 - Phosphorus acids or esters thereof not having P—C bonds, e.g. fosfosal, dichlorvos, malathion
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM (USA)
Inventor
Schunck, Wolf-Hagen
Müller, Dominik
Fischer, Robert
Wallukat, Gerd
Konkel, Anne
Falck, John, Russell
Abstract
The present invention relates to compounds according to general formula (I) which are analogues of epoxymetabolites produced by cytochrome P450 (CYP) enzymes from omega-3 (n-3) polyunsaturated fatty acids (PUFAs). The present invention further relates to compositions containing one or more of these compounds and to the use of these compounds or compositions for the treatment or prevention of conditions and diseases associated with inflammation, proliferation, hypertension, coagulation, immune function, pathologic angiogenesis, heart failure and cardiac arrhythmias.
C07C 309/21 - Sulfonic acids having sulfo groups bound to acyclic carbon atoms of an acyclic unsaturated carbon skeleton containing nitrogen atoms, not being part of nitro or nitroso groups, bound to the carbon skeleton
A61K 31/41 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which is nitrogen, e.g. tetrazole
A61K 31/661 - Phosphorus acids or esters thereof not having P—C bonds, e.g. fosfosal, dichlorvos, malathion
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Lipp, Martin
Oden, Felix
Höpken, Uta
Müller, Gerd
Daumke, Oliver
Marino, Stephen
Olal, Daniel
Abstract
The invention relates to antibodies or antibody fragments that bind CD269 (BCMA), thereby disrupting the interaction between CD269 and its native ligands (BAFF and APRIL), and their use in the treatment of plasma cell-mediated diseases such as multiple myeloma and autoimmune diseases.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
A61P 37/00 - Drugs for immunological or allergic disorders
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Schmetzer, Oliver
Pezzutto, Antonio
Abstract
The present invention relates to synthetic non-antibody-derived CD22-binding peptides for antibody mimetics, as active agents or as targeting agents for immune conjugates.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Klussmann, Enno
Tröger, Jessica
Rosenthal, Walter
Abstract
The present invention relates to compound characterized by a general formula 1, wherein R1 is a aryl or a heteroaryl, and - R2 and R3 independently of each other are hydrogen or a C1-C5 alkyl, but at least one of R2 and R3 is not hydrogen, or together are a C3 or C4 alkyl forming a 5- or 6 membered ring, for use in a method for treating of heart failure, hypertension, cardiac hypertrophy or cancer.
A61K 31/35 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
A61P 9/00 - Drugs for disorders of the cardiovascular system
A61P 9/04 - Inotropic agents, i.e. stimulants of cardiac contractionDrugs for heart failure
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Landthaler, Markus
Munschauer, Mathias
Baltz, Alexander
Abstract
The invention relates to an in vitro method for identifying the sequence of one or more poly(A)+RNA molecules that physically interacts with protein. The present invention provides a method to define the protein-bound transcriptome under any given cellular condition, such as a disease condition or after treatment with any given substance, drug, or other cellular perturbation. The invention also relates to a method for identification of a drug target and a method for the identification of one or more biomarkers, preferably for identification of a panel of biomarkers, for any given medical condition, comprising the method of the invention.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Bimmler, Marion
Karczewski, Peter
Hempel, Petra
Abstract
The invention relates to means for detecting, binding, removing and/or neutralising agonistic antibodies associated with dementia, preferably Alzheimer's disease or vascular dementia.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
FORSCHUNGSVERBUND BERLIN E.V. (Germany)
Inventor
Schäfer, Gesa
Krause, Gerd
Rosenthal, Walter
Milic, Jelena
Rademann, Jörg
Schillinger, Christian
Klussmann, Enno
Abstract
The instant invention relates to compounds characterized by a general formula I and use thereof as a medicament, preferably for prevention or treatment of heart failure (I).
A61K 31/435 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
A61P 9/04 - Inotropic agents, i.e. stimulants of cardiac contractionDrugs for heart failure
24.
NICLOSAMIDE FOR THE TREATMENT OF CANCER METASTASIS
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Stein, Ulrike
Walther, Wolfgang
Sack, Ulrike
Shoemaker, Robert
Scudiero, Dominic
Schlag, Peter M.
Abstract
The invention relates to niclosamide and derivates thereof, which effectively inhibit transcription of the S100A4 gene, resulting in inhibition and/or reduction of S100A4-induced cell motility, invasiveness, metastasis and proliferation of human cancer cells.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Wanker, Erich
Herbst, Martin
Bieschke, Jan
Wagner, Anne
Wiglenda, Thomas
Schmidt, Michael
Böddrich, Annett
Abstract
The present invention relates to compounds useful in the treatment of amyloid diseases, such as Alzheimer's disease. Further, the compounds are useful for imaging amyloid deposits.
A61K 31/538 - 1,4-Oxazines, e.g. morpholine ortho- or peri-condensed with carbocyclic ring systems
C07D 265/38 - [b, e]-condensed with two six-membered rings
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
26.
USE OF APTAMERS IN THERAPY AND/OR DIAGNOSIS OF AUTOIMMUNE DISEASES
The present invention is directed to an aptamer comprising or consisting of the nucleic acid sequence of SEQ ID No. 1, SEQ ID No. 2, SEQ ID No. 3 and/or a nucleic acid sequence being at least 80% identical to one of SEQ ID No. 1, 2 and 3 for use in therapy and/or diagnosis of autoimmune diseases, wherein the autoimmune disease is cardiomyopathy, dilated cardiomyopathy (DCM), peripartum cardiomyopathy (PPCM), idiopathic cardiomyopathy, Chagas' cardiomyopathy, Chagas' megacolon, Chagas' megaesophagus, Chagas' neuropathy, benign prostatic hyperplasia, scleroderma, psoriasis, Raynaud syndrome, pre-eclamsia, kidney allograft rejection, myocarditis, glaucoma, hypertension, pulmonary hypertension, malignant hypertension, and/or Alzheimer's disease.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Schimke, Ingolf
Haberland, Annekathrin
Wallukat, Gerd
Abstract
The present invention is directed to an aptamer comprising or consisting of the nucleic acid sequence of SEQ ID No. 1, SEQ ID No. 2, SEQ ID No. 3 and/or a nucleic acid sequence being at least 80% identical to one of SEQ ID No. 1, 2 and 3 for use in therapy and/or diagnosis of autoimmune diseases, wherein the autoimmune disease is cardiomyopathy, dilated cardiomyopathy (DCM), peripartum cardiomyopathy (PPCM), idiopathic cardiomyopathy, Chagas' cardiomyopathy, Chagas' megacolon, Chagas' megaesophagus, Chagas' neuropathy, benign prostatic hyperplasia, scleroderma, psoriasis, Raynaud syndrome, pre-eclamsia, kidney allograft rejection, myocarditis, glaucoma, hypertension, pulmonary hypertension, malignant hypertension, and/or Alzheimer's disease.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Niendorf, Thoralf
Frauenrath, Tobias
Abstract
A magnetic resonance tomography (MRT) apparatus (1) for the examination of a body (14) comprises parameter acquisition devices (13) for the acquisition of cardiovascular parameters of the body (14) and a control device (15) in communication with the parameter acquisition devices (13) for synchronizing the imaging, wherein the control device (15) is adapted to analyse the data of at least two parameter acquisition devices (13) and to output a control signal based on the analysis.
A61B 5/055 - Detecting, measuring or recording for diagnosis by means of electric currents or magnetic fieldsMeasuring using microwaves or radio waves involving electronic [EMR] or nuclear [NMR] magnetic resonance, e.g. magnetic resonance imaging
G01R 33/567 - Image enhancement or correction, e.g. subtraction or averaging techniques gated by physiological signals
29.
MAGNETIC FIELD APPARATUS AND METHOD OF OPERATING A MAGNETIC FIELD APPARATUS
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
SIEMENS AG (Germany)
Inventor
Frauenrath, Tobias
Niendorf, Thoralf
Renz, Wolfgang
Abstract
A magnetic field apparatus comprises a generator for generating a magnetic field of the magnetic field apparatus and a sensor for measuring the interaction between electrical charge carriers in an object and the magnetic field, wherein the electrical charge carriers and the magnetic field are in relative motion to each other. The magnetic field apparatus may in particular be an MRI apparatus and the sensor may use the magnetohydrodynamic effect for deriving a cardiac trigger signal at any target area positioned off-centre to the heart.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Winter, Lukas
Niendorf, Thoralf
Abstract
A magnetic resonance tomography system (1) for examining a body (2) comprises a sensor (3) for measuring the flow behaviour of a bodily fluid, an MRT apparatus (4) for examining the body (2), which apparatus is set up for an MRT flow measurement or for MR elastography, and a controller (5) for setting a parameter (VENC) of the MRT apparatus (4), which determines imaging, on the basis of the measured flow behaviour.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
FORSCHUNGSVERBUND BERLIN E.V. (Germany)
Inventor
Klussmann, Enno
Rosenthal, Walter
Milic, Jelena
Bergmann, Martin W.
Abstract
The invention relates to N-arylaminomethylenebenzothiophenones of General Formula (I) for use as a drug for the treatment of cardiovascular diseases, wherein E is S, O, or CH2, D is CH or NH, and Ar is a phenyl or naphtyl moiety substituted by an electron-withdrawing group, an unsubstituted heteroaryl residue, or a heteroaryl residue substituted by alkyl or an electron-withdrawing group.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
APTARES AG (Germany)
Inventor
Schimke, Ingolf
Haberland, Annekathrin
Kage, Andreas
Wallukat, Gerd
Dahmen, Claudia
Abstract
The present invention is directed to aptamers which can inhibit the agonistic effect of an antibody specific for the 2nd extracellular loop of human beta-1-adrenergic receptor in a rat cardiomyocyte beating frequency assay with an IC50 of 100 nM or less.
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
C12N 15/115 - Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
33.
BIOMARKERS FOR DETERMINATION OF TEMPORAL PHASE OF ACUTE KIDNEY INJURY
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Schmidt-Ott, Kai
Wuebken, Anne
Abstract
The invention relates to a method for determining the temporal phase of acute kidney injury, comprising obtaining a test sample from a subject and measuring the expression level of at least one biomarker selected from the group comprising Chac1, Birc5 and Angptl7. The invention also relates to a method for determining the early early phase, the late early phase, the severity and/or timing of acute kidney injury via analysis of the biomarkers Chac1, Birc5 and/or Angptl7, in addition to a test kit for carrying out said methods and antibodies directed against Chac1, Birc5 or Angptl7.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Freund, Christian
Schlundt, Andreas
Wieczorek, Marek
Sticht, Jana
Günther, Sebastian
Abstract
The invention relates to a chimeric protein, which comprises an α-chain and ß-chain of a MHC-class II protein, a linker and an epitope of interest, wherein the epitope is linked to the α-chain via the linker.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Hillmeister, Philipp
Buschmann, Ivo
Lenoble, Ferdinand
Gatzke, Nora
Abstract
The present invention relates to bradykinin receptor modulators and pharmaceutical compositions thereof for use as a medicament for modulating collateral blood vessel growth of collateral arteries and/or other blood vessels of pre-existing arterial networks. The bradykinin receptor modulators of arteriogenesis are applicable in the treatment and/or prevention of disorders associated with defective blood flow or blood vessel malformation. A preferred aspect of the invention relates to bradykinin receptor agonists for use as a medicament for the prevention of cardiovascular ischemic disease in a patient at risk thereof. Further, the invention relates to a bradykinin receptor agonist for use in a method for treating a cardiovascular ischemic disease in a patient in need thereof, wherein said cardiovascular ischemic disease is a peripheral limb disease.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Wanker, Erich
Wiglenda, Thomas
Babila, Julius Tachu
Böddrich, Annett
Schmidt, Michael
Schiele, Franziska
Abstract
The present invention relates to compounds suitable as modulators of protein misfolding and/or protein aggregation. The compounds are particularly suitable as inhibitors of amyloid aggregate formation and/or modulators of amyloid surface properties, and/or as activators of degradation or reduction of amyloid aggregates.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61P 43/00 - Drugs for specific purposes, not provided for in groups
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Stein, Ulrike
Herrmann, Pia
Burock, Susen
Schlag, Peter Michael
Abstract
Transcript quantification of the metastasis progressor S100A4 is performed in body fluids. Methods, assays and kits relying on this quantification are disclosed that have diagnostic and/or prognostic applications in colorectal and gastric cancer patients.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Wanker, Erich
Wiglenda, Thomas
Babila, Julius, Tachu
Böddrich, Annett
Schmidt, Michael
Neuendorf, Sandra
Schiele, Franziska
Abstract
The present invention relates to compounds suitable for modulating huntingtin protein processing and useful for treating or preventing huntingtin-related disorders. The invention provides pharmaceutical compositions comprising said compounds and methods of syntheses thereof.
The present invention is directed to a kit-of-parts or composition containing nucleic acid sequences coding for high-avidity, allo-restricted TCR, wherein the TCR are independently directed against the tyrosinase antigen, the melan-A antigen and the survivin antigen. The invention is further directed to a kit-of-parts or composition containing at least three groups of transgenic lymphocytes transformed with vectors coding for TCR against said antigens. Furthermore, the present invention provides a pharmaceutical composition and its use in the treatment of diseases involving malignant cells expressing said tumor-associated antigens. The invention further relates to a nucleic acid molecule coding for a TCR that recognizes the survivin antigen, a TCR encoded thereby and a T cell expressing said TCR. Further, the invention discloses a vector, a cell and a pharmaceutical composition encoding/containing same and their use in the treatment of diseases involving malignant cells expressing survivin.
MAX-DELBRÜCK-CENTRUM FÜR MOLKULARE MEDIZIN (Germany)
Inventor
Buschmann, Ivo
Pries, Axel
Lenoble, Ferdinand
Abstract
A method for determining an arteriovascular condition of a subject having an arterial blood flow is shown. The method involves determining a temporal progression of an instantaneous blood flow condition of the arterial blood flow as well as deriving a slew rate of the temporal progression during an increase of the temporal progression. In addition, an arteriovascular condition indicator device is shown, which comprises: an input for receiving an input signal representing an instantaneous arterial blood flow condition of a subject and a slew rate monitor connected to the input. A corresponding control device for providing an activation signal is also shown. The control device comprises a maximum detector connected to the slew rate monitor. A method for stimulation of arteriogenesis is also shown, wherin a temporal progression of an instantaneous blood flow condition is monitored, a slew rate of the temporal progression is derived, and the maximum of the slew rate is determined. An external pressure is applied repeatedly to the arteriovasculat section in synchronization with the occurrence of the determined maximum.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
JUSTUS-LIEBIG-UNIVERSITÄT GIESSEN (Germany)
Inventor
Wallukat, Gerd
Blaes, Franz
Abstract
The invention relates to nucleic acid molecules encoding peptides which interact with autoantibodies associated with Complex Regional Pain Syndrome, to the peptides themselves, to a pharmaceutical composition comprising said nucleic acid molecules and peptides, and to the use of said peptides - especially in apheresis and in vitro assays- for the treatment and the diagnosis of Complex Regional Pain Syndrome.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
HUMBOLDT-UNIVERSITÄT ZU BERLIN (Germany)
Inventor
Falk, Kirsten
Rötzschke, Olaf
Lucius, Richard
Abstract
The present invention relates to a fusion polypeptide comprising a) at least one fragment of the Plasmodium falciparum S-antigen or a variant thereof, wherein the fragment comprises one or a plurality of Plasmodium falciparum S-antigen repeat unit(s) or variant(s) thereof and b) at least one antigen different from the S-antigen. The present invention also relates to a pharmaceutical composition comprising that fusion polypeptide as well as to the use thereof for the treatment or prevention of an autoimmune disease, an allergic reaction, a transplantation-related complication, e.g. graft rejection or 'graft vs. host disease', and another inflammatory disease and a method for treatment or prevention of said conditions.
The present invention refers to hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB). The invention further refers to corresponding nucleic acids producing these variants, to a gene transfer system for stably introducing nucleic acid(s) into the DNA of a cell by using these hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB) and to transposons used in the inventive gene transfer system, comprising a nucleic acid sequence with flanking repeats (IRs and/or RSDs). Furthermore, applications of these transposase variants, the transpsoson, or the gene transfer system are also disclosed such as gene therapy, insertional mutagenesis, gene discovery (including genome mapping), mobilization of genes, library screening, or functional analysis of genomes in vivo and in vitro. Finally, pharmaceutical compositions and kits are also encompassed.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Izsvak, Zsuzsanna
Ivics, Zoltan
Mates, Lajos
Manoj, Namitha
Judis, Carmen-Anisia
Katzer, Andrea
Abstract
The present invention refers to hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB). The invention further refers to corresponding nucleic acids producing these variants, to a gene transfer system for stably introducing nucleic acid(s) into the DNA of a cell by using these hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB) and to transposons used in the inventive gene transfer system, comprising a nucleic acid sequence with flanking repeats (IRs and/or RSDs). Furthermore, applications of these transposase variants, the transpsoson, or the gene transfer system are also disclosed such as gene therapy, insertional mutagenesis, gene discovery (including genome mapping), mobilization of genes, library screening, or functional analysis of genomes in vivo and in vitro. Finally, pharmaceutical compositions and kits are also encompassed.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
DEUTSCHES HERZZENTRUM BERLIN (Germany)
Inventor
Wallukat, Gerd
Harrer, Thomas
Michael, Dandel
Abstract
The application relates to peptides and nucleic acid molecules encoding peptides which interact with autoantibodies associated with coronary arterial occlusion, angina pectoris, pulmonary hypertension, occlusion of peripheral arterial vessels, Raynaud syndrome and scleroderma, to the peptides themselves, to a pharmaceutical composition comprising said nucleic acid molecules and peptides, and to the use of said peptides - especially in apheresis - for the treatment of coronary arterial occlusion, angina pectoris, pulmonary hypertension, occlusion of peripheral arterial vessels, Raynaud syndrome and scleroderma. The peptides of the application are minimally capable of binding the endothelin-1-receptor and for selected diseases additionally the protease-activated- receptor 1, 2 or 3 or the alphal-adrenergic-receptor.
A61P 9/10 - Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
A61P 37/00 - Drugs for immunological or allergic disorders
C07K 7/00 - Peptides having 5 to 20 amino acids in a fully defined sequenceDerivatives thereof
C07K 14/72 - ReceptorsCell surface antigensCell surface determinants for hormones
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
46.
REMEDY FOR THE TREATMENT OF CARDIO-VASCULAR DISEASES OR DISORDERS
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Bergmann, Martin
Hülsken, Jörg
Birchmeier, Walter
Taketo, Makoto, Mark
Abstract
The present invention relates to the use of an agent for downregulating or inhibiting β-catenin dependent signaling for the treatment of cardiovascular diseases or disorders.
A61K 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
47.
PHARMACEUTICAL PREPARATION FOR TREATING METASTASES
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Zeisig, Reinhardt
Fichtner, Iduna
Wenzel, Jane
Nowak, Christian
Abstract
The invention relates to novel pharmaceutical preparations and their use for the production of drugs for treating metastases, based on liposomes. These preparations are liposomal formulations. According to the invention, these formulations comprise at least one substance having an anti-tumor effect and at least one substance having an anti-coagulation effect, in addition to potentially present further agents. Particularly they are made of base lipids and optionally further components, such as helper lipids, membrane stabilizers, and charge carriers, and may have a modified surface for improving their effectiveness.
A61K 31/685 - Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols one of the hydroxy compounds having nitrogen atoms, e.g. phosphatidylserine, lecithin
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
48.
MEANS FOR INCREASING INTRACELLULAR LEVELS OF PROTOPORPHYRIN IX AND USE THEREOF
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
CHARITE- UNIVERSITÄTSMEDIZIN BERLIN (Germany)
Inventor
Kemmner, Wolfgang
Moesta, Thomas
Schlag, Peter-Michael
Abstract
The present invention is related to a nucleic acid molecule comprising a double-stranded structure, whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and the first stretch is at least partially complementary to a target nucleic acid, and whereby the second strand comprises a second stretch of contiguous nucleotides and whereby said second stretch is at least partially complementary to the first stretch, whereby the first stretch comprises a nucleic acid sequence which is complementary to a nucleotide sequence starting from position 383, 1013 or 1982 of the nucleic acid according to SEQ.ID.No. 1.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
50.
USE OF POL III PROMOTERS FOR CONTROLLED EXPRESSION OF THERAPEUTIC PROTEINS
Max-Delbrück-Centrum für Molekulare Medizin (Germany)
Inventor
Charo, Jehad
Blankenstein, Thomas
Abstract
The present invention relates generally to the field of molecular biology. More specifically, the present invention relates to expression systems for use in modulating the expression of protein coding target genes in vivo or in vitro. More specific, this invention relates to RNA polymerase III-based methods and systems for expression of therapeutic proteins in cells in vivo or in vitro.
The invention relates to nucleic acid molecules encoding peptides which interact with autoantibodies associated with glaucoma, to the peptides themselves, to a pharmaceutical composition comprising said nucleic acid molecules and peptides, and to the use of said peptides - especially in apheresis - for the treatment of glaucoma.
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
FORSCHUNGSVERBUND BERLIN E. V. (Germany)
Inventor
Hellmuth, Klaus
Birchmeier, Walter
Rademann, Jörg
Grosskopf, Stefanie
Abstract
The present invention relates to small molecule protein tyrosine phosphatase inhibitors, especially Shp-2 inhibitors, of formula (I) and/or (II), and to pharmaceutical compositions comprising them. The invention is also directed to the use of said compounds for the treatment of phosphatase-mediated diseases, especially cancer and metastasis. The invention further concerns a method for treating a proliferative disease, a genetic disorder, an autoimmune disease, an angiogenic disorder or cancer in a patient in need of such treatment, comprising administering to said patient a therapeutically effective amount of at least one compound of formula (I) and/or (II).
C07D 417/04 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings directly linked by a ring-member-to-ring- member bond
C07D 231/48 - Oxygen atom in position 3 or 5 and nitrogen atom in position 4 with hydrocarbon radicals attached to said nitrogen atom
A61K 31/427 - Thiazoles not condensed and containing further heterocyclic rings
A61K 31/4152 - 1,2-Diazoles having oxo groups directly attached to the heterocyclic ring, e.g. antipyrine, phenylbutazone, sulfinpyrazone
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
CHARITE UNIVERSITÄTSMEDIZIN BERLIN (Germany)
Inventor
Bärwolff, Dieter
Lindner, Rolf
Munz, Dieter-Ludwig
Michael, Rainer
Abstract
The invention relates to new side chain-fluorinated 3'-deoxythymidine derivatives of the general formula (I) or physiological acceptable esters thereof, wherein R = CH218F; CH18F2; CH2F or CHF2 and Y = H, N3 or Hal. The invention also relates to a simple and efficient method for the preparation of these 3'-deoxythymidines of formula (I) and to the use of the 18F-containing compounds of formula (I) or of a pharmaceutical composition comprising these compounds for the diagnosis of tumors in positron emission tomography (PET).
A61K 31/7072 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN (Germany)
Inventor
Miskey, Csaba
Ivics, Zoltan
Abstract
The present invention refers to a gene transfer system for stably introducing nucleic acid(s) into the DNA of a cell by using a member of the human mariner transposases. The invention further refers to this transposase and to transposons used in the inventive gene transfer system, comprising a nucleic acid sequence with flanking repeats (IRs and/or IR/DRs). Furthermore, applications of this gene transfer system are also disclosed such as gene therapy, insertional mutagenesis, gene discovery (including genome mapping mobilization of genes, library screening, or functional analysis of genomes in vivo and in vitro. Finally, pharmaceutical compositions and kits are also encompassed.