JP7332622B2 - ハンチンチン（ｈｔｔ）の調節のためのジンクフィンガータンパク質組成物 - Google Patents

ハンチンチン（ｈｔｔ）の調節のためのジンクフィンガータンパク質組成物 Download PDF

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JP7332622B2
JP7332622B2 JP2020556850A JP2020556850A JP7332622B2 JP 7332622 B2 JP7332622 B2 JP 7332622B2 JP 2020556850 A JP2020556850 A JP 2020556850A JP 2020556850 A JP2020556850 A JP 2020556850A JP 7332622 B2 JP7332622 B2 JP 7332622B2
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zfp
htt
cells
expression
mice
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スティーブン フローリック，
イグナシオ ムニョス－サンフアン，
エドワード ジェイ． リーバー，
ブライアン ザイトラー，
エイチ． スティーブ ジャン，
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Sangamo Therapeutics Inc
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    • 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
    • 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
    • C07K14/4703—Inhibitors; Suppressors
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
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    • 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
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    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
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  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
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  • Toxicology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Microbiology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2020556850A 2018-04-18 2019-04-17 ハンチンチン（ｈｔｔ）の調節のためのジンクフィンガータンパク質組成物 Active JP7332622B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862659552P 2018-04-18 2018-04-18
US62/659,552 2018-04-18
US201862780605P 2018-12-17 2018-12-17
US62/780,605 2018-12-17
PCT/US2019/027891 WO2019204457A1 (en) 2018-04-18 2019-04-17 Zinc finger protein compositions for modulation of huntingtin (htt)

Publications (3)

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JP2021521775A JP2021521775A (ja) 2021-08-30
JP2021521775A5 JP2021521775A5 (https=) 2022-03-24
JP7332622B2 true JP7332622B2 (ja) 2023-08-23

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US (1) US11421007B2 (https=)
EP (1) EP3781587A4 (https=)
JP (1) JP7332622B2 (https=)
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WO (1) WO2019204457A1 (https=)

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EP3911349A4 (en) * 2019-01-15 2023-01-18 Sangamo Therapeutics, Inc. HTT REPRESSORS AND THEIR USES
EP4716741A2 (en) * 2023-05-23 2026-04-01 Flagship Labs 114, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
CN121729237A (zh) 2023-06-30 2026-03-24 武田药品工业株式会社 Htt阻遏物及其用途
WO2025151707A1 (en) 2024-01-11 2025-07-17 Entrada Therapeutics, Inc. Method for preparing multimers of phosphorodiamidate morpholino oligomers
EP4696705A1 (en) * 2024-08-13 2026-02-18 Fondazione Telethon ETS Engineered transcription repressors and uses thereof
CN118649252B (zh) * 2024-08-21 2024-11-05 核工业四一六医院 Zfp36l2基因在治疗和/或预防辐射损伤中的应用

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US20190322711A1 (en) 2019-10-24
CN112313246A (zh) 2021-02-02
EP3781587A1 (en) 2021-02-24
CN112313246B (zh) 2025-05-13
EP3781587A4 (en) 2022-03-30
JP2021521775A (ja) 2021-08-30
US11421007B2 (en) 2022-08-23
WO2019204457A1 (en) 2019-10-24

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