Abstract: This invention relates to methods for altering the splicing of mRNA in cells. In particular, this invention also relates to methods for increasing the ratio of wild type to misspliced forms of mRNA and corresponding encoded proteins in cells possessing a mutant gene encoding either the i) misspliced mRNA corresponding to the mutant protein or ii) a component in the splicing machinery responsible for processing the misspliced mRNA. In addition, this invention relates to treating individuals having a disorder associated with a misspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, by administering to such an individual a cytokinin such as kinetin.
Type:
Grant
Filed:
August 31, 2012
Date of Patent:
May 20, 2014
Assignee:
The General Hospital Corporation
Inventors:
Susan A. Slaugenhaupt, James F. Gusella
Abstract: Provided herein are methods and compositions for treating a subject suffering from a deficiency in arylsulfatase A in the CNS. The methods include systemic administration of a bifunctional fusion antibody comprising an antibody to a human insulin receptor and an arylsulfatase A.
Abstract: The invention is directed to methods of treatment of Alzheimer's disease and other tauopathies, via the administration of antibodies having specificity to abnormal forms of tau protein, the antibodies showing no binding and/or reactivity to a normal tau protein and being administered under conditions and in amounts effective to prevent or treat Alzheimer's disease or other tauopathies. In certain embodiments, the antibodies are selective for soluble truncated tau protein truncated at (i) its C-terminus after the glutamic acid residue Glu391, or (ii) at the aspartic acid residue Asp421, or (iii) at its N-terminus at the aspartic acid residue Asp13, or (iv) a combination of (i)-(iii). Further aspects of the invention are directed to the administration of an immunogen comprising an abnormal tau, preferably a soluble truncated tau.
Abstract: Antibodies selective for pathological tau dimers and/or prefibrillar pathological tau oligomers, immunogenic peptides and epitopes of these antibodies, hydridomas producing these antibodies, uses of these antibodies, immunogenic peptides and epitopes in preparation of pharmaceutical compositions for the treatment of tauopathies, and uses of these antibodies, immunogenic peptides, epitopes and pharmaceutical compositions in the treatment of tauopathies are described. Also described are uses of these antibodies, immunogenic peptides, epitopes in diagnosis and monitoring of tauopathies.
Type:
Grant
Filed:
April 27, 2012
Date of Patent:
April 15, 2014
Assignees:
Northwestern University, Intellect Neurosciences Inc.
Inventors:
Lester I. Binder, Daniel G. Chain, Yifan Fu, Kristina Patterson
Abstract: The present invention provides methods of treating or preventing a substance-related disorder using selective delta opioid receptor-1 (DOP-R1) agonists, delta opioid receptor-2 (DOP-R2) antagonists, and/or mu opioid receptor (MOP-R) antagonists, or a pharmaceutically acceptable salt, solvate, hydrate, or prodrug thereof. The methods provided herein further comprise administering a therapeutically effective amount of a combination of a DOP-R1 agonist and a DOP-R2 antagonist. The methods also comprise administering a therapeutically effective amount of a combination of a DOP-R1 agonist and an MOP-R antagonist. The methods provided herein further comprise administering a therapeutically effective amount of a combination of a DOP-R1 agonist and a DOP-R2 antagonist and a MOP-R antagonist. The invention also relates to compositions containing the same. The invention also relates to methods of determining delta opioid receptor specificity of candidate agents.
Type:
Grant
Filed:
May 1, 2009
Date of Patent:
April 8, 2014
Assignee:
The Regents of the University of California
Inventors:
Howard L. Fields, Jennifer M. Mitchell, Elyssa B. Margolis
Abstract: Methods are provided for the treatment of subjects with cognitive or neuropsychiatric impairment induced by substance addiction and for increasing cognitive function in a subject with substance addiction. In some embodiments, the methods include administering to the subject a therapeutically effective amount of a major histocompatibility complex (MHC) molecule including covalently linked first, second, and third domains; wherein the first domain is an MHC class II ?1 domain and the second domain is an MHC class II ?1 domain; or wherein the first domain is an MHC class I ?1 domain and the second domain is an MHC class I ?2 domain; and wherein the third domain is covalently linked to the first domain and comprises an antigen of the central or peripheral nervous system.
Type:
Grant
Filed:
January 30, 2012
Date of Patent:
April 1, 2014
Assignees:
Oregon Health & Science University, The United States Government as Represented by the Department of Veterans Affairs
Inventors:
Jennifer Loftis, Marilyn Huckans, Arthur A. Vandenbark
Abstract: The invention provides methods of treating stroke and related conditions exacerbated by fever and/or hyperglycemia by administering peptides or peptidomimetics that inhibit binding of NMDAR 2B to PSD-95 to a patient.
Abstract: This invention discloses CN2097-like compositions that facilitate the induction of long-term potentiation (LTP). In one embodiment the method comprises inducing long-term potentiation in a subject by the method of administering a therapeutically effective dose of a CN2097-like compound.
Abstract: An isolated, pure homogeneous population of mammalian astrocyte restricted precursor cells which is CD44 immunoreactive and which generate astrocytes but not oligodendrocytes is provided. Methods for isolating and using these mammalian astrocyte restricted precursor cells are also provided.
Type:
Grant
Filed:
April 30, 2009
Date of Patent:
March 18, 2014
Assignees:
University of Utah, The Government of the United States of America
Inventors:
Mahendra S. Rao, Tahmina Mujtaba, Yuan Yuan Wu, Ying Liu
Abstract: A method of detecting MSDX Complex-1, the method introducing a first antibody to a sample to create an antibody-sample mixture, wherein the first antibody is specific for one of fibrinogen, fibronectin, or fibulin-1, the first antibody having a label molecule; providing a well coated with a second antibody, the second antibody is specific for one of fibrinogen, fibronectin, or fibulin-1; introducing the antibody-sample mixture to the well; and introducing a substrate to the antibody-sample mixture in the well, wherein the label molecule and the substrate interact to provide a signal, wherein when the signal is detected then MSDX Complex-1 is detected.
Abstract: Methods and therapeutic agents are disclosed for treating neurodegenerative disorders by depletion of CD8 positive T cells by using antibodies, FAb fragments of antibodies or similar agents that sequester, neutralize or deplete the CD8+ cytotoxic T cells.
Type:
Grant
Filed:
February 23, 2012
Date of Patent:
March 18, 2014
Assignee:
ALS Therapy Development Institute
Inventors:
Steven Perrin, John Lincecum, Alan Gill, Fernando Vieira
Abstract: The present invention is related to methods and pharmaceutical compositions for the therapeutic and diagnostic use in the treatment of diseases and disorders which are caused by or associated with neurofibrillary tangles. In particular, the invention relates to pharmaceutical composition comprising an antigenic peptide, particularly an antigenic phospho-peptide mimicking a major pathological phospho-epitope of protein tau, for the therapeutic and diagnostic use in the treatment of tauopathies including Alzheimer's Disease.
Type:
Grant
Filed:
April 1, 2010
Date of Patent:
February 11, 2014
Assignees:
Katholieke Universiteit Leuven, AC Immune S.A.
Inventors:
Andrea Pfeifer, Andreas Muhs, Fred Van Leuven, Maria Pihlgren
Abstract: Candidate compounds for use in neuro-protection and repair in neurological disorders involving Tau dysfunction (including Alzheimer's disease) are identified from a direct interaction between proteins FKBP52 and Tau. The method for screening a drug for the prevention and treatment of neurological disorders involving Tau dysfunction includes determining the ability of a candidate compound, to modulate binding between a Tau polypeptide and a FKBP52 polypeptide, and selecting positively the candidate compound that modulates binding.
Type:
Grant
Filed:
September 24, 2010
Date of Patent:
February 11, 2014
Assignee:
Institut National de la Sante et de la Recherche Medicale (INSERM)
Abstract: Novel compounds which are antagonists or inverse agonists at one or more of the opioid receptors, pharmaceutical compositions containing them, to processes for their preparation.
Type:
Grant
Filed:
August 8, 2007
Date of Patent:
January 21, 2014
Assignee:
GlaxoSmithKline LLC
Inventors:
David John Cowan, Andrew Lamont Larkin, Cunyu Zhang, David Lee Musso, Gary Martin Green, Rodolfo Cadilla, Paul Kenneth Spearing, Michael Joseph Bishop, Jason Daniel Speake
Abstract: Antibodies and fragments thereof have high affinity for human ?-synuclein protofibrils and low binding of ?-synuclein monomers, wherein the antibodies or fragments have specified Complementarity Determining Region (CDR) sequences. Compositions comprise such an antibody or fragment and methods of detecting ?-synuclein protofibrils use such an antibody or fragment. In further embodiments, methods of preventing, delaying onset of or treating a neurodegenerative disorder with ?-synuclein pathology comprise administering such an antibody or fragment, and such an antibody or fragment is used in the manufacture of a pharmaceutical composition for treatment of a neurodegenerative disorder with ?-synuclein pathology. Such an antibody or fragment is used in the diagnosis or monitoring of the development of a neurodegenerative disorder with ?-synuclein pathology, and in methods for reducing or inhibiting ?-synuclein aggregation by administration of such an antibody or fragment.
Type:
Grant
Filed:
February 25, 2011
Date of Patent:
January 21, 2014
Assignee:
BioArctic Neuroscience AB
Inventors:
Eva Nordström, Alex Kasrayan, Monica Ekberg, Valentina Screpanti Sundquist, Lars Lannfelt, Mats Holmquist
Abstract: An object of the present invention is to provide a safe and effective method for enhancing an immune response and a medicament for preventing or treating Alzheimer disease comprising amyloid ? peptide that induces an enhanced immune response. An amyloid ? peptide or a portion thereof with addition or insertion of cysteine and a method for enhancing an immune response using the peptide or a method for enhancing an immune response using the peptide together with an adjuvant. A medicament for preventing or treating Alzheimer disease comprising an amyloid ? peptide or a portion thereof that induces an enhanced immune response. A DNA vaccine, that may have the same effect, comprising the gene encoding an amyloid ? peptide or a portion thereof that induces an enhanced immune response with addition or insertion of cysteine.
Type:
Grant
Filed:
August 29, 2011
Date of Patent:
January 7, 2014
Assignee:
The Chemo-Sero-Therapeutic Research Institute
Abstract: The present invention relates to methods for restoring fast axonal transport in a cell which expresses a pathological synuclein protein and for treating a synucleinopathy using a Protein Kinase C mu or Src-Family Tyrosine Kinase inhibitor.
Type:
Grant
Filed:
April 9, 2008
Date of Patent:
December 31, 2013
Assignee:
The Board of Trustees of the University of Illinois
Inventors:
Scott Thomas Brady, Gerardo Andres Morfini
Abstract: This invention relates, to a method for promoting CNS axon regeneration, comprising (1) inhibiting the expression or activity in a neuron of one or more of the members of the Krüppel-like transcription factor (KLF) family that suppress axon growth (e.g., KLF 1, 2, 3, 4, 5, 9,12, 13, 14, 15 and/or 16), and/or (2) stimulating the expression or activity in a neuron of one or more of the members of the KLF family that promote axon growth (e.g., KLF 6 and/or 7).
Type:
Grant
Filed:
September 7, 2010
Date of Patent:
December 17, 2013
Assignee:
University of Miami
Inventors:
Jeffrey L. Goldberg, Vance P. Lemmon, John Bixby, Darcie Moore, Murray Blackmore
Abstract: The present invention is related to methods and compositions for the therapeutic and diagnostic use in the treatment of diseases and disorders which are caused by or associated with amyloid or amyloid-like proteins including amyloidosis. In particular, the present invention provides novel methods and compositions for eliciting a highly specific and highly effective immune response in an organism, but particularly within an animal, particularly a mammal or a human, which is capable of preventing or alleviating amyloidosis, or the symptoms associated with amyloidosis, a group of diseases and disorders associated with amyloid plaque formation including secondary amyloidosis and age-related amyloidosis including, but not limited to, neurological disorders such as Alzheimer's Disease (AD), including diseases or conditions characterized by a loss of cognitive memory capacity such as, for example, mild cognitive impairment (MCI).
Abstract: The invention relates to compositions and methods to confer protection on neurons. Peptides derived from the NAPVSIPQ (SEQ ID NO:4) peptide and including branched amino acids, such as alpha-aminoisobutyric acid, are included. Also included are methods of preventing and treating neurodegenerative disorders and damage caused by neurotoxic substances.