AU2018255293B2 - Regulation of RAN translation by PKR and eIF2a-P pathways - Google Patents

Regulation of RAN translation by PKR and eIF2a-P pathways Download PDF

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AU2018255293B2
AU2018255293B2 AU2018255293A AU2018255293A AU2018255293B2 AU 2018255293 B2 AU2018255293 B2 AU 2018255293B2 AU 2018255293 A AU2018255293 A AU 2018255293A AU 2018255293 A AU2018255293 A AU 2018255293A AU 2018255293 B2 AU2018255293 B2 AU 2018255293B2
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poly
pkr
protein
ran
spinocerebellar ataxia
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AU2018255293A1 (en
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Laura Ranum
Tao Zu
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University of Florida Research Foundation Inc
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    • A61K31/155—Amidines (), e.g. guanidine (H2N—C(=NH)—NH2), isourea (N=C(OH)—NH2), isothiourea (—N=C(SH)—NH2)
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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/45—Transferases (2)
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
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    • C12N15/09—Recombinant DNA-technology
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
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AU2018255293A 2017-04-17 2018-04-17 Regulation of RAN translation by PKR and eIF2a-P pathways Active AU2018255293B2 (en)

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US201762486424P 2017-04-17 2017-04-17
US62/486,424 2017-04-17
US201762563588P 2017-09-26 2017-09-26
US62/563,588 2017-09-26
PCT/US2018/028015 WO2018195110A1 (en) 2017-04-17 2018-04-17 Regolation of ran translation by pkr and eif2a-p pathways

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AU2018255293B2 true AU2018255293B2 (en) 2024-03-07

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EP (1) EP3612190A4 (https=)
JP (2) JP7526455B2 (https=)
AU (2) AU2018255293B2 (https=)
CA (1) CA3060250A1 (https=)
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Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014159247A1 (en) 2013-03-14 2014-10-02 University Of Florida Research Foundation, Inc. Di-amino acid repeat-containing proteins associated with als
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
US10940161B2 (en) 2016-04-04 2021-03-09 University Of Florida Research Foundation, Incorporated Manipulation of EIF3 to modulate repeat associated non-ATG (RAN) translation
WO2018195110A1 (en) 2017-04-17 2018-10-25 University Of Florida Research Foundation, Incorporated Regolation of ran translation by pkr and eif2a-p pathways
EP3688020A4 (en) 2017-09-25 2021-07-14 University of Florida Research Foundation, Incorporated IMMUNOASSAYS FOR DETECTION OF RAN PROTEINS
AU2018342105B2 (en) * 2017-09-26 2023-11-16 University Of Florida Research Foundation, Incorporated Use of metformin and analogs thereof to reduce ran protein levels in the treatment of neurological disorders
CN109504684B (zh) * 2018-12-20 2021-07-27 山西大学 Ran基因及其dsRNA在害虫防治中的应用
EP3962518A1 (en) 2019-05-02 2022-03-09 Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. (DZNE) Immunogen for preventing or treating familial frontotemporal dementia (ftd) and/or amyotrophic lateral sclerosis (als)
WO2021055880A1 (en) * 2019-09-20 2021-03-25 University Of Florida Researchfoundation, Incorporated Detection of antibodies against ran proteins from serum and tissue lysates
EP4440560A1 (en) * 2021-12-01 2024-10-09 University Of Florida Research Foundation, Incorporated Small molecule inhibitors of repeat associated non-aug (ran) translation and combination therapies

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070036760A1 (en) * 2003-09-30 2007-02-15 The Trutees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
WO2010132982A1 (en) * 2009-05-18 2010-11-25 Ottawa Hospital Research Institute Treatment of muscle disease characterized by insulin resistance
US20160096800A1 (en) * 2013-03-15 2016-04-07 The Regents Of The University Of California Modulators of the eif2alpha pathway
WO2018035408A1 (en) * 2016-08-18 2018-02-22 Ovid Therapeutics Inc. Methods of treating developmental disorders with biguanides

Family Cites Families (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5011912A (en) 1986-12-19 1991-04-30 Immunex Corporation Hybridoma and monoclonal antibody for use in an immunoaffinity purification system
ATE247129T1 (de) 1995-04-11 2003-08-15 Merck & Co Inc Biologisches verfahren zur herstellung von verbindungen auf der basis der dipeptiden
CA2283379A1 (en) * 1997-03-05 1998-09-11 Michael G. Katze Novel screening methods to identify agents that selectively inhibit hepatitis c virus replication
EP1000084A4 (en) 1997-07-08 2003-03-26 Human Genome Sciences Inc 123 HUMAN SECRETED PROTEINS
US6362007B1 (en) 1997-08-29 2002-03-26 Innogenetics N.V. Methylated, SMD homologous peptides, reactive with the antibodies from sera of living beings affected with systemic lupus erythematosus
US6413783B1 (en) 1997-09-18 2002-07-02 Meso Scale Technologies, Llc Assay sonication apparatus and methodology
US6436703B1 (en) 2000-03-31 2002-08-20 Hyseq, Inc. Nucleic acids and polypeptides
JP2004520803A (ja) 2000-04-21 2004-07-15 コリクサ コーポレイション 尋常性挫瘡の治療および診断のための組成物および方法
JP2004518437A (ja) 2000-10-25 2004-06-24 ディアデクサス インコーポレーテッド 肺特異的遺伝子およびタンパク質に関する組成物および方法
EP1349934A2 (en) 2000-11-20 2003-10-08 Diadexus, Inc. Compositions and methods relating to breast specific genes and proteins
US7528227B2 (en) 2004-03-23 2009-05-05 Yissum Research Development Company Of The Hebrew University Of Jerusalem Histone H2A peptide derivatives and uses thereof
US7163943B2 (en) 2001-09-21 2007-01-16 Reddy Us Therapeutics, Inc. Methods and compositions of novel triazine compounds
US7314974B2 (en) 2002-02-21 2008-01-01 Monsanto Technology, Llc Expression of microbial proteins in plants for production of plants with improved properties
US7393642B2 (en) 2003-07-17 2008-07-01 Chicago Community Foundation Methods and primers for diagnosing idiopathic congenital central hypoventilation syndrome
AU2005222776A1 (en) 2003-12-31 2005-09-29 Genimmune N.V. Inducing cellular immune responses to human papillomavirus using peptide and nucleic acid compositions
US7481997B1 (en) 2004-02-12 2009-01-27 Montana State University Snow mountain virus genome sequence, virus-like particles and methods of use
US20090075900A1 (en) 2004-04-08 2009-03-19 Sadakazu Therapeutic agent for motor neuron disease
EP1844148A2 (en) 2005-01-31 2007-10-17 University of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
WO2006133194A2 (en) 2005-06-06 2006-12-14 Vgx Pharmaceuticals, Inc Methods for treating viral infection with oral or injectible drug solution
US20080248099A1 (en) 2005-11-11 2008-10-09 Ishii Douglas N Method for Treating Disease or Disorder of Adult Central Nervous System Associated with Tissue Shrinkage or Atrophy by Administration of Insulin
PL2082225T3 (pl) 2006-09-20 2014-06-30 Univ Belfast Sposób określania czy komórka nowotworowa ma potencjał inwazyjny i/lub metastatyczny oraz środki modulujące złośliwą transformację
US20080188457A1 (en) 2007-02-02 2008-08-07 Braincells, Inc. Modulation of Neurogenesis with Biguanides and GSK3-beta Agents
US20090148866A1 (en) 2007-05-18 2009-06-11 Abbott Laboratories Antibodies and Improved Test Sample Handling Methods for Use in Assays for Myeloperoxidase
EP2164865A2 (en) 2007-06-13 2010-03-24 Hochschule Mannheim Compounds for the modulation of huntingtin aggregation, methods and means for identifying such compounds
US20090143418A1 (en) 2007-11-30 2009-06-04 The Regents Of The University Of Michigan Compositions and Methods for Preventing and Treating Hair Growth Cycle-Related Conditions
GB0809821D0 (en) 2008-05-30 2008-07-09 Univ Aberdeen Treatment and diagnosis of behavioural disorders
BRPI0923761B1 (pt) 2008-12-30 2019-08-13 3M Innovative Properties Co aerossol adesivo e névoa de aerossol
TW201034684A (en) 2009-02-18 2010-10-01 Genentech Inc Method for inhibiting neurodegeneration
CA2757354A1 (en) 2009-04-02 2010-10-07 Laura P.W. Ranum Nucleotide repeat expansion-associated polypeptides and uses thereof
US20130053426A1 (en) 2009-04-17 2013-02-28 Yiqi Seow Composition For Delivery Of Genetic Material
WO2011028912A2 (en) 2009-09-03 2011-03-10 University Of Tennessee Research Foundation A biomarker for neurodegeneration in neurological disease
KR101133243B1 (ko) 2009-10-29 2012-04-06 국립암센터 암의 진단 및 치료를 위한 ｅＩＦ３ｍ의 용도
EP2552435A1 (en) 2010-04-02 2013-02-06 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and compositions comprising ampk activator (metformin/troglitazone) for the treatment of myotonic dystrophy type 1 (dm1)
EP2647706B1 (en) 2010-11-30 2023-05-17 Chugai Seiyaku Kabushiki Kaisha Antigen-binding molecule capable of binding to plurality of antigen molecules repeatedly
US9896729B2 (en) 2011-08-31 2018-02-20 The University Of Manchester Method for diagnosing a neurodegenerative disease
US9587014B2 (en) 2011-10-28 2017-03-07 Biogen International Neuroscience Gmbh TDP-43 specific binding molecules
WO2013130824A1 (en) 2012-02-29 2013-09-06 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
KR101417272B1 (ko) 2012-05-15 2014-07-14 경상대학교산학협력단 메트포민 및/또는 타이모퀴논을 포함하는 임신중 알코올 노출에 의한 신경질환 치료 또는 예방용 약학적 조성물
US20140100282A1 (en) 2012-10-10 2014-04-10 Patrick S L Wong Intranasal administration of pharmaceutical agents for treatment of neurological diseases
WO2014114303A1 (en) 2013-01-22 2014-07-31 Deutsches Zentrum Für Neurodegenerative Erkrankungen Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion
WO2014114660A1 (en) 2013-01-22 2014-07-31 Deutsches Zentrum Für Neurodegenerative Erkrankungen E.V. (Dzne) Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion
EP2948471A4 (en) 2013-01-24 2016-08-10 Mayo Foundation METHOD AND MATERIALS FOR DETECTING POSITIVE FRONTOTEMPORAL PAPER DEGENERATION BY C9ORF72 HEXANUCLEOTIDE REPEAT EXPANSION OR AMYOTROPHATE LATERAL SCLEROSIS BY C9ORF72 HEXANUCLEOTIDE REPEAT EXPANSION
WO2014159247A1 (en) 2013-03-14 2014-10-02 University Of Florida Research Foundation, Inc. Di-amino acid repeat-containing proteins associated with als
WO2014186746A1 (en) 2013-05-16 2014-11-20 University Of Florida Research Foundation, Inc. HAIRPIN mRNA ELEMENTS AND METHODS FOR THE REGULATION OF PROTEIN TRANSLATION
EP2837390A1 (en) 2013-08-15 2015-02-18 Universitäts-Kinderspital beider Basel Combined Pharmaceutical Preparation for Use in Treating Neuromuscular Disorders
CA2990161A1 (en) 2014-08-13 2016-02-18 The Scripps Research Institute Treatment of c9ftd/als by targeting rna expanded repeat sequences
JP6508996B2 (ja) 2015-03-24 2019-05-08 東ソー株式会社 β−ＡＮＰによる心不全の検出方法
WO2016191241A1 (en) 2015-05-27 2016-12-01 Mmc & Company Llc Dual target mitochondrial impinging pharmaceutical compositions affecting mitochondrial redox state and methods of treatment
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
FI3356827T3 (fi) 2015-10-02 2024-01-03 Univ Wuerzburg J Maximilians GDF-15 diagnostisena markkerina immuunivasteen vapauttajilla annetun hoidon hoitovasteen ennustamiseksi
US10940161B2 (en) 2016-04-04 2021-03-09 University Of Florida Research Foundation, Incorporated Manipulation of EIF3 to modulate repeat associated non-ATG (RAN) translation
WO2018195110A1 (en) 2017-04-17 2018-10-25 University Of Florida Research Foundation, Incorporated Regolation of ran translation by pkr and eif2a-p pathways
EP3688020A4 (en) 2017-09-25 2021-07-14 University of Florida Research Foundation, Incorporated IMMUNOASSAYS FOR DETECTION OF RAN PROTEINS
AU2018342105B2 (en) 2017-09-26 2023-11-16 University Of Florida Research Foundation, Incorporated Use of metformin and analogs thereof to reduce ran protein levels in the treatment of neurological disorders
EP3962518A1 (en) 2019-05-02 2022-03-09 Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. (DZNE) Immunogen for preventing or treating familial frontotemporal dementia (ftd) and/or amyotrophic lateral sclerosis (als)
EP3994159A4 (en) 2019-07-05 2023-08-09 University of Florida Research Foundation, Incorporated METHODS OF TREATING NEUROLOGICAL DISEASES ASSOCIATED WITH THE RAN PROTEIN
WO2021055880A1 (en) 2019-09-20 2021-03-25 University Of Florida Researchfoundation, Incorporated Detection of antibodies against ran proteins from serum and tissue lysates
WO2021061537A1 (en) 2019-09-23 2021-04-01 University Of Florida Research Foundation, Incorporated Vaccine therapy for ran protein diseases
EP4041756A4 (en) 2019-10-10 2023-11-01 University Of Florida Research Foundation, Incorporated RAN PROTEINS AS BIOMARKERS IN CAG/CTG EXPANSION DISORDERS
WO2021231887A1 (en) 2020-05-15 2021-11-18 University Of Florida Research Foundation, Incorporated Compositions and methods of detection of pre-symptomatic als
EP4440560A1 (en) 2021-12-01 2024-10-09 University Of Florida Research Foundation, Incorporated Small molecule inhibitors of repeat associated non-aug (ran) translation and combination therapies

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070036760A1 (en) * 2003-09-30 2007-02-15 The Trutees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
WO2010132982A1 (en) * 2009-05-18 2010-11-25 Ottawa Hospital Research Institute Treatment of muscle disease characterized by insulin resistance
US20160096800A1 (en) * 2013-03-15 2016-04-07 The Regents Of The University Of California Modulators of the eif2alpha pathway
WO2018035408A1 (en) * 2016-08-18 2018-02-22 Ovid Therapeutics Inc. Methods of treating developmental disorders with biguanides

Non-Patent Citations (9)

* Cited by examiner, † Cited by third party
Title
Abstract 266 'Old friends on new paths: metformin as an early phase treatmentin Huntington’s Disease?', Medizinische Genetik, (2016-03-04), vol. 28, no. 1, pages 84 - 232, doi: 10.1007/S11825-016-0083-5 *
Bando, Y. et al., Neurochemistry International, (2005), vol. 46, no. 1, pages 11-18, doi:10.1016/j.neuint.2004.07.005. *
Benkirane, M. et al., 'Oncogenic potential of TAR RNA binding protein TRBP and its regulatory interaction with RNA-dependent protein kinase PKR', The EMBO Journal, (1997), vol. 16, no. 3, pages 611-624, doi: 10.1093/emboj/16.3.611 *
Guerra, S. et al., The Journal of Biological Chemistry, vol. 281, no. 27, pages 18734-18745, doi: 10.1074/jbc.M511983200. *
Jin, J. et al., Neuromolecular Medicine, (2016-05-25), vol. 18, no. 4, pages 581 - 592, doi: 10.1007/S12017-016-8412-Z. *
Ma, T. et al., 'Metformin therapy in a transgenic mouse model of Huntington's disease', Neuroscience Letters, (2006-11-29), vol. 411, no. 2, pages 98 - 103, doi: 10.1016/J.NEULET.2006.10.039. *
Peel, A. et al., 'Double-stranded RNA-dependent protein kinase, PKR, binds preferentially to Huntington's disease (HD) transcripts and is activated in HD tissue', Human Molecular Genetics, (2001), vol. 10, no. 15, pages 1531-1538. *
Welnowska, E., 'Translation of Viral mRNA without Active eIF2: The Case of Picornaviruses', PLOS ONE, (2011-07-14), vol. 6, no. 7, pages 1 - 13. *
Wojciechowska, M. et al., Nucleic Acids Research, (2014-10-29), vol. 42, no. 19, pages 11849 - 11864, doi: 10.1093/nar/gku794. *

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