EP3601541A4 - Mutiertes fus-modell für als - Google Patents

Mutiertes fus-modell für als Download PDF

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Publication number
EP3601541A4
EP3601541A4 EP17902030.0A EP17902030A EP3601541A4 EP 3601541 A4 EP3601541 A4 EP 3601541A4 EP 17902030 A EP17902030 A EP 17902030A EP 3601541 A4 EP3601541 A4 EP 3601541A4
Authority
EP
European Patent Office
Prior art keywords
fus
muted
als
model
muted fus
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.)
Withdrawn
Application number
EP17902030.0A
Other languages
English (en)
French (fr)
Other versions
EP3601541A1 (de
Inventor
Xue ZHANG
Yichang Jia
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.)
Tsinghua University
Original Assignee
Tsinghua 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.)
Filing date
Publication date
Application filed by Tsinghua University filed Critical Tsinghua University
Publication of EP3601541A1 publication Critical patent/EP3601541A1/de
Publication of EP3601541A4 publication Critical patent/EP3601541A4/de
Withdrawn legal-status Critical Current

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Classifications

    • 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
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • 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
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
    • G01N33/5058—Neurological cells
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5082—Supracellular entities, e.g. tissue, organisms
    • G01N33/5088—Supracellular entities, e.g. tissue, organisms of vertebrates
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0356—Animal model for processes and diseases of the central nervous system, e.g. stress, learning, schizophrenia, pain, epilepsy
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/2835—Movement disorders, e.g. Parkinson, Huntington, Tourette

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Hematology (AREA)
  • Biochemistry (AREA)
  • Urology & Nephrology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Analytical Chemistry (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Food Science & Technology (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Environmental Sciences (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
EP17902030.0A 2017-03-22 2017-03-22 Mutiertes fus-modell für als Withdrawn EP3601541A4 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/CN2017/077708 WO2018170794A1 (en) 2017-03-22 2017-03-22 Mutant fus model for als

Publications (2)

Publication Number Publication Date
EP3601541A1 EP3601541A1 (de) 2020-02-05
EP3601541A4 true EP3601541A4 (de) 2020-10-28

Family

ID=63586119

Family Applications (1)

Application Number Title Priority Date Filing Date
EP17902030.0A Withdrawn EP3601541A4 (de) 2017-03-22 2017-03-22 Mutiertes fus-modell für als

Country Status (3)

Country Link
EP (1) EP3601541A4 (de)
CN (1) CN110770336B (de)
WO (1) WO2018170794A1 (de)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113196055B (zh) * 2018-10-15 2025-06-27 马克斯·普朗克科学促进协会 用于治疗疾病的化合物及其筛选方法
EP3921650A1 (de) 2019-02-08 2021-12-15 Dewpoint Therapeutics, Inc. Verfahren zur charakterisierung von kondensatassoziierten eigenschaften von verbindungen und deren verwendungen
CA3140651A1 (en) * 2019-05-15 2020-11-19 Whitehead Institute For Biomedical Research Methods of characterizing and utilizing agent-condensate interactions
CN114729941A (zh) * 2019-09-18 2022-07-08 露点治疗公司 筛选凝聚物相关特异性的方法及其用途
CN116042619B (zh) * 2022-12-19 2023-09-08 神济昌华(北京)生物科技有限公司 用于构建人源FUS基因敲入的ALS果蝇模型的gRNA组合及其应用

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2731643C (en) * 2008-07-22 2020-05-05 The General Hospital Corporation Fus/tls-based compounds and methods for diagnosis, treatment and prevention of amyotrophic lateral sclerosis and related motor neuron diseases
US20140349938A1 (en) * 2011-06-03 2014-11-27 President And Fellows Of Harvard College Methods of diagnosing and treating amyotrophic lateral sclerosis
CN106350521B (zh) * 2016-08-24 2019-11-12 中国科学院生物物理研究所 一种als患者特异性运动神经元的制备方法

Non-Patent Citations (8)

* Cited by examiner, † Cited by third party
Title
"Current Protocols in Pharmacology", 1 June 2015, JOHN WILEY & SONS, INC., Hoboken, NJ, USA, ISBN: 978-0-471-14175-4, article THOMAS PHILIPS ET AL: "Rodent Models of Amyotrophic Lateral Sclerosis : Rodent Models of ALS", pages: 5.67.1 - 5.67.21, XP055549612, DOI: 10.1002/0471141755.ph0567s69 *
"Program and Abstracts of the 13th Transgenic Technology Meeting (TT2016) ED - Murray James D; Ross Pablo J; Van Eenennaam Alison; Maga Elizabeth A", TRANSGENIC RESEARCH, SPRINGER NETHERLANDS, NL, vol. 25, no. 2, 16 February 2016 (2016-02-16), pages 195 - 270, XP035933679, ISSN: 0962-8819, [retrieved on 20160216], DOI: 10.1007/S11248-016-9936-6 *
BARBER S C ET AL: "An in vitro screening cascade to identify neuroprotective antioxidants in ALS", FREE RADICAL BIOLOGY AND MEDICINE, ELSEVIER INC, US, vol. 46, no. 8, 15 April 2009 (2009-04-15), pages 1127 - 1138, XP026586639, ISSN: 0891-5849, [retrieved on 20090130], DOI: 10.1016/J.FREERADBIOMED.2009.01.019 *
CAO HUANG ET AL: "FUS Transgenic Rats Develop the Phenotypes of Amyotrophic Lateral Sclerosis and Frontotemporal Lobar Degeneration", PLOS GENETICS, vol. 7, no. 3, 3 March 2011 (2011-03-03), pages e1002011, XP055729988, ISSN: 1553-7390, DOI: 10.1371/journal.pgen.1002011 *
EDISON T LIU ET AL: ": future of the mouse as a model system for the human condition", EMBO REPORTS, vol. 18, no. 2, 24 January 2017 (2017-01-24), GB, pages 187 - 193, XP055730069, ISSN: 1469-221X, DOI: 10.15252/embr.201643717 *
HAIYAN QIU ET AL: "ALS-associated mutation FUS-R521C causes DNA damage and RNA splicing defects", THE JOURNAL OF CLINICAL INVESTIGATION, vol. 124, no. 3, 10 February 2014 (2014-02-10), GB, pages 981 - 999, XP055539623, ISSN: 0021-9738, DOI: 10.1172/JCI72723 *
JACQUELINE C MITCHELL ET AL: "Overexpression of human wild-type FUS causes progressive motor neuron degeneration in an age- and dose-dependent fashion", ACTA NEUROPATHOLOGICA, SPRINGER, BERLIN, DE, vol. 125, no. 2, 9 September 2012 (2012-09-09), pages 273 - 288, XP035167366, ISSN: 1432-0533, DOI: 10.1007/S00401-012-1043-Z *
MARCIE A GLICKSMAN: "The preclinical discovery of amyotrophic lateral sclerosis drugs", EXPERT OPINION ON DRUG DISCOVERY, vol. 6, no. 11, 25 October 2011 (2011-10-25), London, GB, pages 1127 - 1138, XP055730112, ISSN: 1746-0441, DOI: 10.1517/17460441.2011.628654 *

Also Published As

Publication number Publication date
CN110770336A (zh) 2020-02-07
EP3601541A1 (de) 2020-02-05
CN110770336B (zh) 2023-09-12
WO2018170794A1 (en) 2018-09-27

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