CA3060250A1 - Regulation of ran protein translation by pkr and eif2a-p pathways - Google Patents

Regulation of ran protein translation by pkr and eif2a-p pathways Download PDF

Info

Publication number
CA3060250A1
CA3060250A1 CA3060250A CA3060250A CA3060250A1 CA 3060250 A1 CA3060250 A1 CA 3060250A1 CA 3060250 A CA3060250 A CA 3060250A CA 3060250 A CA3060250 A CA 3060250A CA 3060250 A1 CA3060250 A1 CA 3060250A1
Authority
CA
Canada
Prior art keywords
protein
pkr
ran
poly
compound
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.)
Pending
Application number
CA3060250A
Other languages
English (en)
French (fr)
Inventor
Laura Ranum
Tao Zu
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 CA3060250A1 publication Critical patent/CA3060250A1/en
Pending legal-status Critical Current

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)
CA3060250A 2017-04-17 2018-04-17 Regulation of ran protein translation by pkr and eif2a-p pathways Pending CA3060250A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3060250A1 true CA3060250A1 (en) 2018-10-25

Family

ID=63856032

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3060250A Pending CA3060250A1 (en) 2017-04-17 2018-04-17 Regulation of ran protein translation by pkr and eif2a-p pathways

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

Family Cites Families (65)

* 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
EP2345731B1 (en) * 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
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
CA2762351A1 (en) * 2009-05-18 2010-11-25 Ottawa Hospital Research Institute Treatment of muscle disease characterized by insulin resistance
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

Also Published As

Publication number Publication date
JP7526455B2 (ja) 2024-08-01
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
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
US12473545B2 (en) Regulation of RAN translation by PKR and eIF2α-P pathways
US10653693B2 (en) PRMT5 inhibitors and uses thereof
US12030869B2 (en) 5-membered heteroaryl carboxamide compounds for treatment of HBV
AU2018342105B2 (en) Use of metformin and analogs thereof to reduce ran protein levels in the treatment of neurological disorders
US20250163028A1 (en) Methods and compounds for the treatment of genetic disease
US11578032B2 (en) Derivatives of sobetirome
US9284309B2 (en) Di- and tri-heteroaryl derivatives as inhibitors of protein aggregation
Gosset et al. Prionoids in amyotrophic lateral sclerosis
US9845320B2 (en) Benzothiazole or benzoxazole compounds as sumo activators
US12128105B2 (en) Molecular degraders of extracellular proteins
WO2023064326A1 (en) Methods of treating diseases associated with senescent cell accumulation
WO2023051805A1 (zh) 卤代氧化苯砷化合物及其应用
US10953036B2 (en) Compositions and methods of modulating HIF-2A to improve muscle generation and repair
US20250164489A1 (en) Ran proteins in sporadic amyotrophic lateral sclerosis
US20100016221A1 (en) Method of degrading protein by chaperone-mediated autophagy
US11345705B2 (en) Substituted pyrazolo[1,5-a]pyrimidines as apelin receptor agonists
WO2019154195A1 (zh) 神经退行性疾病的治疗药物及其应用
KR101306157B1 (ko) 사이클로펜타펩타이드를 유효성분으로 포함하는 종양의 예방 또는 치료용 조성물
CN106860445A (zh) 血竭素、血竭素衍生物或其可用盐在制备糖尿病药物中的应用

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20230417

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20240906

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250131

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 7TH ANNIV.) - STANDARD

Year of fee payment: 7

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250411

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250411

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20250507

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20250507

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-P146 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: BSL VERIFIED - NO DEFECTS

Effective date: 20250704

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20250717

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20251117

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20251118

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20251118

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20251118