WO2016134246A3 - Compositions et méthodes de traitement de maladies conformationnelles des protéines - Google Patents

Compositions et méthodes de traitement de maladies conformationnelles des protéines Download PDF

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Publication number
WO2016134246A3
WO2016134246A3 PCT/US2016/018657 US2016018657W WO2016134246A3 WO 2016134246 A3 WO2016134246 A3 WO 2016134246A3 US 2016018657 W US2016018657 W US 2016018657W WO 2016134246 A3 WO2016134246 A3 WO 2016134246A3
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WO
WIPO (PCT)
Prior art keywords
methods
protein conformational
conformational diseases
compositions
treating protein
Prior art date
Application number
PCT/US2016/018657
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English (en)
Other versions
WO2016134246A2 (fr
Inventor
Jiou Wang
Goran PERIZ
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The Johns Hopkins University
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.)
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Publication date
Application filed by The Johns Hopkins University filed Critical The Johns Hopkins University
Publication of WO2016134246A2 publication Critical patent/WO2016134246A2/fr
Publication of WO2016134246A3 publication Critical patent/WO2016134246A3/fr

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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/033—Rearing or breeding invertebrates; New breeds of invertebrates
    • A01K67/0333—Genetically modified invertebrates, e.g. transgenic, polyploid
    • A01K67/0335—Genetically modified worms
    • A01K67/0336—Genetically modified Nematodes, e.g. Caenorhabditis elegans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y603/00—Ligases forming carbon-nitrogen bonds (6.3)
    • C12Y603/02—Acid—amino-acid ligases (peptide synthases)(6.3.2)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/05—Animals modified by non-integrating nucleic acids, e.g. antisense, RNAi, morpholino, episomal vector, for non-therapeutic purpose
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/20—Animals treated with compounds which are neither proteins nor nucleic acids
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Environmental Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Hospice & Palliative Care (AREA)
  • Immunology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente invention concerne le domaine des maladies conformationnelles des protéines. Plus spécifiquement, la présente invention concerne des compositions et des méthodes de traitement de maladies conformationnelles des protéines, y compris la sclérose latérale amyotrophique (SLA). Dans un mode de réalisation, un procédé comprend l'étape d'administration, à un patient, d'une quantité efficace d'un inhibiteur Ube4B et d'un inhibiteur LSD1. Dans un autre mode de réalisation, le procédé comprend en outre l'étape d'administration d'un agoniste de p53.
PCT/US2016/018657 2015-02-20 2016-02-19 Compositions et méthodes de traitement de maladies conformationnelles des protéines WO2016134246A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562118503P 2015-02-20 2015-02-20
US62/118,503 2015-02-20

Publications (2)

Publication Number Publication Date
WO2016134246A2 WO2016134246A2 (fr) 2016-08-25
WO2016134246A3 true WO2016134246A3 (fr) 2016-11-03

Family

ID=56689486

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2016/018657 WO2016134246A2 (fr) 2015-02-20 2016-02-19 Compositions et méthodes de traitement de maladies conformationnelles des protéines

Country Status (1)

Country Link
WO (1) WO2016134246A2 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117180410A (zh) * 2022-06-01 2023-12-08 浙江大学 UbV.E4B蛋白的医药用途、药物组合物

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012042042A1 (fr) * 2010-09-30 2012-04-05 Oryzon Genomics S.A. Inhibiteurs sélectifs de lsd1 et lsd1/mao-b double pour moduler des maladies associées à des altérations de conformation de protéine

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012042042A1 (fr) * 2010-09-30 2012-04-05 Oryzon Genomics S.A. Inhibiteurs sélectifs de lsd1 et lsd1/mao-b double pour moduler des maladies associées à des altérations de conformation de protéine

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
FISKUS, W. ET AL.: "Highly effective combination of LSD1 (KDMIA) antagonist and pan-histone deacetylase inhibitor against human AML cells", LEUKEMIA, vol. 28, 2014, pages 1 - 10, XP055326198 *
PERIZ, G. ET AL.: "Regulation of protein quality control by UBE4B and LSD1 through p53-mediated transcription", PLOS BIOLOGY, vol. 13, no. 4, 2 April 2015 (2015-04-02), pages 1 - 29, XP055326199 *
SUN, G. ET AL.: "Histone demethylase LSD1 regulates neural stem cell proliferation", MOLECULAR AND CELLULAR BIOLOGY, vol. 30, no. 8, 2010, pages 1997 - 2005, XP055326195 *
ZHANG, X.-F. ET AL.: "Expression and prognostic role of ubiquitination fact or E4B in primary hepatocellular carcinoma", MOLECULAR CARCINOGENESIS, vol. 55, pages 64 - 76, XP055326197 *

Also Published As

Publication number Publication date
WO2016134246A2 (fr) 2016-08-25

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