JP7542912B2 - Httリプレッサーおよびその使用 - Google Patents

Httリプレッサーおよびその使用 Download PDF

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Publication number
JP7542912B2
JP7542912B2 JP2021540305A JP2021540305A JP7542912B2 JP 7542912 B2 JP7542912 B2 JP 7542912B2 JP 2021540305 A JP2021540305 A JP 2021540305A JP 2021540305 A JP2021540305 A JP 2021540305A JP 7542912 B2 JP7542912 B2 JP 7542912B2
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zfp
mhtt
cells
protein
gene
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Japanese (ja)
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JP2022517988A5 (https=
JP2022517988A (ja
JPWO2020150338A5 (https=
Inventor
ガレン キャリー,
マシュー チオッコ,
ヴィヴィアン チョイ,
ブライアン フェリス,
スティーブン フローリック,
デブラ クラッテ,
ジェフリー ミラー,
デイビッド パッション,
エドワード リーバー,
ブライアン ザイトラー,
レイ ジャン,
エイチ. スティーブ ジャン,
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Takeda Pharmaceutical Co Ltd
Sangamo Therapeutics Inc
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Takeda Pharmaceutical Co Ltd
Sangamo Therapeutics Inc
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4703Inhibitors; Suppressors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021540305A 2019-01-15 2020-01-15 Httリプレッサーおよびその使用 Active JP7542912B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962792701P 2019-01-15 2019-01-15
US62/792,701 2019-01-15
PCT/US2020/013661 WO2020150338A1 (en) 2019-01-15 2020-01-15 Htt repressors and uses thereof

Publications (4)

Publication Number Publication Date
JP2022517988A JP2022517988A (ja) 2022-03-11
JP2022517988A5 JP2022517988A5 (https=) 2023-01-25
JPWO2020150338A5 JPWO2020150338A5 (https=) 2023-01-25
JP7542912B2 true JP7542912B2 (ja) 2024-09-02

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Family Applications (1)

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JP2021540305A Active JP7542912B2 (ja) 2019-01-15 2020-01-15 Httリプレッサーおよびその使用

Country Status (5)

Country Link
US (1) US20220064237A1 (https=)
EP (1) EP3911349A4 (https=)
JP (1) JP7542912B2 (https=)
CN (1) CN113301909B (https=)
WO (1) WO2020150338A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019204457A1 (en) 2018-04-18 2019-10-24 Sangamo Therapeutics, Inc. Zinc finger protein compositions for modulation of huntingtin (htt)
CN115487315B (zh) * 2022-04-20 2023-08-04 暨南大学 治疗亨廷顿病的药物
WO2025004001A1 (en) * 2023-06-30 2025-01-02 Takeda Pharmaceutical Company Limited Htt repressors and uses thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013500045A (ja) 2009-07-28 2013-01-07 サンガモ バイオサイエンシーズ, インコーポレイテッド トリヌクレオチド反復疾患を治療するための方法および組成物
JP2015509958A (ja) 2012-02-29 2015-04-02 サンガモ バイオサイエンシーズ, インコーポレイテッド ハンチントン病を治療するための方法および組成物
JP2018532428A (ja) 2015-09-23 2018-11-08 サンガモ セラピューティクス, インコーポレイテッド Httリプレッサー及びその使用
JP2021521775A (ja) 2018-04-18 2021-08-30 サンガモ セラピューティクス, インコーポレイテッド 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

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7888121B2 (en) * 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) * 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
JP5266210B2 (ja) * 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
WO2015070212A1 (en) * 2013-11-11 2015-05-14 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
CN106459894B (zh) * 2014-03-18 2020-02-18 桑格摩生物科学股份有限公司 用于调控锌指蛋白表达的方法和组合物
RU2691102C2 (ru) * 2014-05-08 2019-06-11 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
GB2544270A (en) * 2015-11-05 2017-05-17 Fundació Centre De Regulació Genòmica Nucleic acids, peptides and methods

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013500045A (ja) 2009-07-28 2013-01-07 サンガモ バイオサイエンシーズ, インコーポレイテッド トリヌクレオチド反復疾患を治療するための方法および組成物
JP2015509958A (ja) 2012-02-29 2015-04-02 サンガモ バイオサイエンシーズ, インコーポレイテッド ハンチントン病を治療するための方法および組成物
JP2018532428A (ja) 2015-09-23 2018-11-08 サンガモ セラピューティクス, インコーポレイテッド Httリプレッサー及びその使用
JP2021521775A (ja) 2018-04-18 2021-08-30 サンガモ セラピューティクス, インコーポレイテッド 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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Gabriela Ecco, A tale of domestication: the endovirome, its polydactyl controllers and the species-specificity of human biology,Development.,2017年,144,2719-29
Zeitler, Bryan,Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease,Nature Medicine,2019年,25,1131-42

Also Published As

Publication number Publication date
EP3911349A1 (en) 2021-11-24
WO2020150338A1 (en) 2020-07-23
EP3911349A4 (en) 2023-01-18
US20220064237A1 (en) 2022-03-03
CN113301909B (zh) 2025-01-28
JP2022517988A (ja) 2022-03-11
CN113301909A (zh) 2021-08-24

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