CA2731643C - Fus/tls-based compounds and methods for diagnosis, treatment and prevention of amyotrophic lateral sclerosis and related motor neuron diseases - Google Patents

Fus/tls-based compounds and methods for diagnosis, treatment and prevention of amyotrophic lateral sclerosis and related motor neuron diseases Download PDF

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CA2731643C
CA2731643C CA2731643A CA2731643A CA2731643C CA 2731643 C CA2731643 C CA 2731643C CA 2731643 A CA2731643 A CA 2731643A CA 2731643 A CA2731643 A CA 2731643A CA 2731643 C CA2731643 C CA 2731643C
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fus
tls
seq
nucleic acid
als
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French (fr)
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CA2731643A1 (en
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Thomas J. Kwiatkowski
Robert H. Brown
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General Hospital Corp
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General Hospital Corp
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • 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
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6834—Enzymatic or biochemical coupling of nucleic acids to a solid phase
    • C12Q1/6837—Enzymatic or biochemical coupling of nucleic acids to a solid phase using probe arrays or probe chips
    • 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
    • G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
    • 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 nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/136—Screening for pharmacological compounds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/172—Haplotypes
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Pathology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Neurology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Neurosurgery (AREA)
  • Food Science & Technology (AREA)
  • General Physics & Mathematics (AREA)
  • Oncology (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pain & Pain Management (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Orthopedic Medicine & Surgery (AREA)
CA2731643A 2008-07-22 2009-07-21 Fus/tls-based compounds and methods for diagnosis, treatment and prevention of amyotrophic lateral sclerosis and related motor neuron diseases Active CA2731643C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US13568908P 2008-07-22 2008-07-22
US61/135,689 2008-07-22
PCT/US2009/004205 WO2010011283A2 (en) 2008-07-22 2009-07-21 Fus/tls-based compounds and methods for diagnosis, treatment and prevention of amyotrophic lateral sclerosis and related motor neuron diseases

Publications (2)

Publication Number Publication Date
CA2731643A1 CA2731643A1 (en) 2010-01-28
CA2731643C true CA2731643C (en) 2020-05-05

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CA2731643A Active CA2731643C (en) 2008-07-22 2009-07-21 Fus/tls-based compounds and methods for diagnosis, treatment and prevention of amyotrophic lateral sclerosis and related motor neuron diseases

Country Status (4)

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US (3) US9150860B2 (OSRAM)
JP (1) JP5643200B2 (OSRAM)
CA (1) CA2731643C (OSRAM)
WO (1) WO2010011283A2 (OSRAM)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5643200B2 (ja) 2008-07-22 2014-12-17 ザ ジェネラル ホスピタル コーポレイション 筋萎縮性側索硬化症および関連する運動ニューロン疾患の診断、処置および予防のための、ｆｕｓ／ｔｌｓベースの化合物および方法
JP6342627B2 (ja) * 2013-08-02 2018-06-13 国立大学法人 東京大学 筋萎縮性側索硬化症の新規病因遺伝子
EP3573637A4 (en) 2017-01-27 2020-11-11 Vanderbilt University INHIBITORS OF NUCLEAR TRANSLOCATION OF FUS PROTEIN ENABLING THE PREVENTION OF FIBROSIS
CN110753557B (zh) * 2017-03-22 2024-03-22 神济昌华(北京)生物科技有限公司 Trim72通过泛素化fus蛋白突变体作为als的潜在治疗性靶标
CN110770336B (zh) * 2017-03-22 2023-09-12 神济昌华(北京)生物科技有限公司 用于als的突变型fus模型
JP7295584B2 (ja) * 2018-11-09 2023-06-21 スンチョニャン ユニヴァーシティ インダストリー アカデミー コーペレーション ファウンデーション 神経系退行性疾患診断用マーカーおよび治療用組成物
EP3892283A1 (en) * 2020-04-09 2021-10-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Nucleic acids encoding human fus protein and use in the treatment of amyotrophic lateral sclerosis (als)
US20240229025A1 (en) * 2020-09-09 2024-07-11 Ohara Pharmaceutical Co., Ltd. siRNA AND USE THEREOF
WO2022082199A1 (en) * 2020-10-16 2022-04-21 Pluripotent Diagnostics Corp. Method for detecting amyotrophic lateral sclerosis
US20250002944A1 (en) * 2021-10-05 2025-01-02 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glad Allele specific editing to treat fus-induced neurodegeneration
US20250101420A1 (en) * 2022-03-08 2025-03-27 Ohara Pharmaceutical Co., Ltd. MODIFIED siRNA FOR SELECTIVELY INHIBITING EXPRESSION OF MUTANT FUS
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 (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6582908B2 (en) * 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
US5843641A (en) 1993-02-26 1998-12-01 Massachusetts Institute Of Technology Methods for the daignosis, of familial amyotrophic lateral sclerosis
JP2002306179A (ja) 2001-04-16 2002-10-22 Japan Science & Technology Corp Ａｌｓ２ｃｒ６遺伝子と、２型筋萎縮性側索硬化症の診断方法
CA2521999A1 (en) * 2002-12-20 2004-09-02 Biotrove, Inc. Assay apparatus and method using microfluidic arrays
WO2004108899A2 (en) 2003-06-04 2004-12-16 The Government Of The United States As Represented By The Secretary Of The Department Of Health And Human Services, Centers For Disease Control And Prevention Pni microarray and uses
US20070009899A1 (en) * 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
WO2006034573A1 (en) * 2004-09-27 2006-04-06 Med Biogene Inc. Hematological cancer profiling system
US20070202537A1 (en) 2005-12-05 2007-08-30 Prosetta Corporation Biomarkers for als
AU2008205312B2 (en) 2007-01-08 2013-11-21 Annji Pharmaceutical Co., Ltd. Compounds with (substituted phenyl)-propenal moiety, their derivatives, biological activity, and uses thereof
JP5643200B2 (ja) 2008-07-22 2014-12-17 ザ ジェネラル ホスピタル コーポレイション 筋萎縮性側索硬化症および関連する運動ニューロン疾患の診断、処置および予防のための、ｆｕｓ／ｔｌｓベースの化合物および方法

Also Published As

Publication number Publication date
WO2010011283A2 (en) 2010-01-28
JP5643200B2 (ja) 2014-12-17
US20160177389A1 (en) 2016-06-23
WO2010011283A3 (en) 2010-05-14
CA2731643A1 (en) 2010-01-28
JP2011528903A (ja) 2011-12-01
US11041204B2 (en) 2021-06-22
US20120046341A1 (en) 2012-02-23
US20210388444A1 (en) 2021-12-16
US9150860B2 (en) 2015-10-06

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