WO2016030899A8 - Methods of treating amyotrophic lateral scleroses - Google Patents

Methods of treating amyotrophic lateral scleroses Download PDF

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Publication number
WO2016030899A8
WO2016030899A8 PCT/IL2015/050867 IL2015050867W WO2016030899A8 WO 2016030899 A8 WO2016030899 A8 WO 2016030899A8 IL 2015050867 W IL2015050867 W IL 2015050867W WO 2016030899 A8 WO2016030899 A8 WO 2016030899A8
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WO
WIPO (PCT)
Prior art keywords
methods
amyotrophic lateral
treating amyotrophic
treating
scleroses
Prior art date
Application number
PCT/IL2015/050867
Other languages
French (fr)
Other versions
WO2016030899A1 (en
Inventor
Eran Hornstein
Tal Yardeni
Original Assignee
Yeda Research And Development Co. Ltd.
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 Yeda Research And Development Co. Ltd. filed Critical Yeda Research And Development Co. Ltd.
Publication of WO2016030899A1 publication Critical patent/WO2016030899A1/en
Publication of WO2016030899A8 publication Critical patent/WO2016030899A8/en

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    • 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
    • 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
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Provided are methods and pharmaceutical compositions for treating Amyotrophic lateral scleroses (ALS) in a human subject in need thereof, by administering to the subject a pharmaceutical composition comprising as an active ingredient an agent capable of downregulating an activity or expression level of miR-9, thereby treating the ALS in the human subject in need thereof.
PCT/IL2015/050867 2014-08-28 2015-08-27 Methods of treating amyotrophic lateral scleroses WO2016030899A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462042800P 2014-08-28 2014-08-28
US62/042,800 2014-08-28

Publications (2)

Publication Number Publication Date
WO2016030899A1 WO2016030899A1 (en) 2016-03-03
WO2016030899A8 true WO2016030899A8 (en) 2016-03-24

Family

ID=54261052

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IL2015/050867 WO2016030899A1 (en) 2014-08-28 2015-08-27 Methods of treating amyotrophic lateral scleroses

Country Status (1)

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WO (1) WO2016030899A1 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3478313B1 (en) 2016-06-29 2022-05-04 CRISPR Therapeutics AG Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders
EP4048803A4 (en) 2019-10-24 2024-11-13 Integrated Dna Tech Inc Modified double-stranded donor templates
US20220025365A1 (en) * 2020-07-23 2022-01-27 Integrated Dna Technologies, Inc. METHODS FOR NOMINATION OF NUCLEASE ON-/OFF-TARGET EDITING LOCATIONS, DESIGNATED "CTL-seq" (CRISPR Tag Linear-seq)
CN114045334A (en) * 2021-09-26 2022-02-15 四川大学华西医院 Marker combination, detection reagent and kit for diagnosing ALS and application thereof

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