JP7526455B2 - ＰＫＲおよびｅＩＦ２Ａ－Ｐ経路によるＲＡＮタンパク質翻訳の調節 - Google Patents

ＰＫＲおよびｅＩＦ２Ａ－Ｐ経路によるＲＡＮタンパク質翻訳の調節 Download PDF

Info

Publication number
JP7526455B2
JP7526455B2 JP2019556606A JP2019556606A JP7526455B2 JP 7526455 B2 JP7526455 B2 JP 7526455B2 JP 2019556606 A JP2019556606 A JP 2019556606A JP 2019556606 A JP2019556606 A JP 2019556606A JP 7526455 B2 JP7526455 B2 JP 7526455B2
Authority
JP
Japan
Prior art keywords
pkr
protein
ran
poly
rna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2019556606A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020516674A (ja
JP2020516674A5 (https=
Inventor
ラヌム，ラウラ
ズ，タオ
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Florida Research Foundation Inc
Original Assignee
University of Florida Research Foundation Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of Florida Research Foundation Inc filed Critical University of Florida Research Foundation Inc
Publication of JP2020516674A publication Critical patent/JP2020516674A/ja
Publication of JP2020516674A5 publication Critical patent/JP2020516674A5/ja
Priority to JP2024025160A priority Critical patent/JP2024081635A/ja
Application granted granted Critical
Publication of JP7526455B2 publication Critical patent/JP7526455B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/13—Amines
    • A61K31/155—Amidines (), e.g. guanidine (H2N—C(=NH)—NH2), isourea (N=C(OH)—NH2), isothiourea (—N=C(SH)—NH2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/01037—Protein kinase (2.7.1.37)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2019556606A 2017-04-17 2018-04-17 ＰＫＲおよびｅＩＦ２Ａ－Ｐ経路によるＲＡＮタンパク質翻訳の調節 Active JP7526455B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024025160A JP2024081635A (ja) 2017-04-17 2024-02-22 ＰＫＲおよびｅＩＦ２Ａ－Ｐ経路によるＲＡＮタンパク質翻訳の調節

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024025160A Division JP2024081635A (ja) 2017-04-17 2024-02-22 ＰＫＲおよびｅＩＦ２Ａ－Ｐ経路によるＲＡＮタンパク質翻訳の調節

Publications (3)

Publication Number Publication Date
JP2020516674A JP2020516674A (ja) 2020-06-11
JP2020516674A5 JP2020516674A5 (https=) 2021-05-27
JP7526455B2 true JP7526455B2 (ja) 2024-08-01

Family

ID=63856032

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019556606A Active JP7526455B2 (ja) 2017-04-17 2018-04-17 ＰＫＲおよびｅＩＦ２Ａ－Ｐ経路によるＲＡＮタンパク質翻訳の調節
JP2024025160A Pending JP2024081635A (ja) 2017-04-17 2024-02-22 ＰＫＲおよびｅＩＦ２Ａ－Ｐ経路によるＲＡＮタンパク質翻訳の調節

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024025160A Pending JP2024081635A (ja) 2017-04-17 2024-02-22 ＰＫＲおよびｅＩＦ２Ａ－Ｐ経路によるＲＡＮタンパク質翻訳の調節

Country Status (6)

Country Link
US (3) US11345911B2 (https=)
EP (1) EP3612190A4 (https=)
JP (2) JP7526455B2 (https=)
AU (2) AU2018255293B2 (https=)
CA (1) CA3060250A1 (https=)
WO (1) WO2018195110A1 (https=)

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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007507223A (ja) 2003-09-30 2007-03-29 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）の同源系統群、配列、それらを含有するベクターおよびそれらの用途
WO2010132982A1 (en) 2009-05-18 2010-11-25 Ottawa Hospital Research Institute Treatment of muscle disease characterized by insulin resistance

Family Cites Families (63)

* 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
JP6806562B2 (ja) 2013-03-15 2021-01-06 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア ｅＩＦ２α経路の調節因子
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
CN109715144A (zh) 2016-08-18 2019-05-03 奥维德医疗公司 使用双胍类治疗发育障碍的方法
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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007507223A (ja) 2003-09-30 2007-03-29 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）の同源系統群、配列、それらを含有するベクターおよびそれらの用途
WO2010132982A1 (en) 2009-05-18 2010-11-25 Ottawa Hospital Research Institute Treatment of muscle disease characterized by insulin resistance

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Gene, 2014, Vol.536, pp.90-96
Neuromol. Med., 2016, Vol.18, pp.581-592
Neuroscience Letters, 2007, Vol.411, pp.98-103
PLOS ONE, 2014, Vol.9, No.3, Article No.e90803, pp.1-10

Also Published As

Publication number Publication date
JP2020516674A (ja) 2020-06-11
WO2018195110A8 (en) 2018-11-29
US20230002753A1 (en) 2023-01-05
EP3612190A4 (en) 2021-01-20
EP3612190A1 (en) 2020-02-26
CA3060250A1 (en) 2018-10-25
US11345911B2 (en) 2022-05-31
US12473545B2 (en) 2025-11-18
US20260103700A1 (en) 2026-04-16
JP2024081635A (ja) 2024-06-18
US20200140846A1 (en) 2020-05-07
AU2024201065A1 (en) 2024-03-07
AU2018255293B2 (en) 2024-03-07
WO2018195110A1 (en) 2018-10-25
AU2018255293A1 (en) 2019-11-28

Similar Documents

Publication Publication Date Title
JP7526455B2 (ja) ＰＫＲおよびｅＩＦ２Ａ－Ｐ経路によるＲＡＮタンパク質翻訳の調節
US11339136B2 (en) Compounds and compositions for treating conditions associated with NLRP activity
US11447460B2 (en) Compounds and compositions for treating conditions associated with NLRP activity
US11578032B2 (en) Derivatives of sobetirome
US11337941B2 (en) Inhibitors and their uses
US9284309B2 (en) Di- and tri-heteroaryl derivatives as inhibitors of protein aggregation
Gosset et al. Prionoids in amyotrophic lateral sclerosis
CN107074752A (zh) 苯亚甲基胍衍生物在治疗蛋白质构象紊乱方面的新型治疗用途
US20250057868A1 (en) Compositions and methods of modulating hif-2a to improve muscle generation and repair
US20150218148A1 (en) Benzothiazole Or Benzoxazole Compounds As Sumo Activators
Shi et al. Compound Danshen dripping pill effectively alleviates cGAS-STING-triggered diseases by disrupting STING-TBK1 interaction
US20100016221A1 (en) Method of degrading protein by chaperone-mediated autophagy
JP2022515570A (ja) インフルエンザ及び合併症リスク因子を有する対象における置換多環式ピリドン誘導体及びそのプロドラッグを用いたインフルエンザの処置
US20230159439A1 (en) Arylamides and methods of use thereof
WO2019154195A1 (zh) 神经退行性疾病的治疗药物及其应用
US20250340537A1 (en) Pharmaceutically active compounds, compositions, and methods for modulating pendrin
Tan et al. Lithium co-crystals and an additional neuropsychiatric agent for treatment of neuropsychiatric disorders

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210414

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210414

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220524

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220815

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221124

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230301

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230601

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230801

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20231024

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240222

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20240222

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20240422

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20240701

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20240712

R150 Certificate of patent or registration of utility model

Ref document number: 7526455

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150