JP2023511908A - タウ発現抑制のためのジンクフィンガータンパク質転写因子 - Google Patents

タウ発現抑制のためのジンクフィンガータンパク質転写因子 Download PDF

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JP2023511908A
JP2023511908A JP2022544353A JP2022544353A JP2023511908A JP 2023511908 A JP2023511908 A JP 2023511908A JP 2022544353 A JP2022544353 A JP 2022544353A JP 2022544353 A JP2022544353 A JP 2022544353A JP 2023511908 A JP2023511908 A JP 2023511908A
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zfp
fusion protein
tau
protein
sequence
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Japanese (ja)
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JP2023511908A5 (https=
Inventor
ザイトラー，ブライアン
ハタミ，アサ
ジャン，レイ
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • 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
    • C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • 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
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Management, Administration, Business Operations System, And Electronic Commerce (AREA)
  • Immunology (AREA)
JP2022544353A 2020-01-22 2021-01-22 タウ発現抑制のためのジンクフィンガータンパク質転写因子 Pending JP2023511908A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062964501P 2020-01-22 2020-01-22
US62/964,501 2020-01-22
PCT/US2021/014780 WO2021151012A1 (en) 2020-01-22 2021-01-22 Zinc finger protein transcription factors for repressing tau expression

Publications (2)

Publication Number Publication Date
JP2023511908A true JP2023511908A (ja) 2023-03-23
JP2023511908A5 JP2023511908A5 (https=) 2024-02-09

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JP2022544353A Pending JP2023511908A (ja) 2020-01-22 2021-01-22 タウ発現抑制のためのジンクフィンガータンパク質転写因子

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Country Link
US (1) US20230242602A1 (https=)
EP (1) EP4093754A1 (https=)
JP (1) JP2023511908A (https=)
KR (1) KR20220131273A (https=)
CN (1) CN115210251A (https=)
AR (1) AR121118A1 (https=)
AU (1) AU2021209699A1 (https=)
BR (1) BR112022014160A2 (https=)
CA (1) CA3168805A1 (https=)
IL (1) IL294535A (https=)
MX (1) MX2022008953A (https=)
TW (1) TW202134288A (https=)
UY (1) UY39036A (https=)
WO (1) WO2021151012A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL266862B2 (en) 2016-12-01 2024-01-01 Sangamo Therapeutics Inc Tau modulators and methods and preparations for their administration
WO2020072677A1 (en) 2018-10-02 2020-04-09 Sangamo Therapeutics, Inc. Methods and compositions for modulation of tau proteins
AU2020356962A1 (en) * 2019-10-02 2022-04-14 Sangamo Therapeutics, Inc. Zinc finger protein transcription factors for repressing alpha-synuclein expression
WO2025193848A1 (en) * 2024-03-13 2025-09-18 Sangamo Therapeutics, Inc. Delivery of tau repressors using the blood-brain barrier penetrant capsid

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020500526A (ja) * 2016-12-01 2020-01-16 サンガモ セラピューティクス， インコーポレイテッド タウモジュレーターならびにその送達のための方法および組成物

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DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
ES2341926T3 (es) 1998-03-02 2010-06-29 Massachusetts Institute Of Technology Poliproteinas con dedos de cinc que tienen enlazadores mejorados.
ATE355368T1 (de) 2000-01-24 2006-03-15 Gendaq Ltd Nucleinsäure bindende polypeptide gekennzeichnet durch flexible linker verbundene nucleinsäuredomäne
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Ｃｎｎについての亜鉛フィンガー結合ドメイン
US9394531B2 (en) 2008-05-28 2016-07-19 Sangamo Biosciences, Inc. Compositions for linking DNA-binding domains and cleavage domains
HRP20200254T1 (hr) 2010-05-03 2020-05-29 Sangamo Therapeutics, Inc. Pripravci za povezivanje modula cink fingera
CA3131284C (en) 2013-08-28 2023-09-19 David Paschon Compositions for linking dna-binding domains and cleavage domains
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
WO2017011556A1 (en) * 2015-07-13 2017-01-19 The General Hospital Corporation Rare phosphorylated high molecular weight (hmw) tau species that are involved in neuronal uptake and propagation and applications thereof
KR20180101442A (ko) 2016-02-02 2018-09-12 상가모 테라퓨틱스, 인코포레이티드 Dna-결합 도메인 및 절단 도메인을 연결시키기 위한 조성물
KR102455249B1 (ko) 2016-08-24 2022-10-17 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
WO2020072677A1 (en) 2018-10-02 2020-04-09 Sangamo Therapeutics, Inc. Methods and compositions for modulation of tau proteins
EP3861130A4 (en) 2018-10-02 2022-08-03 Sangamo Therapeutics, Inc. GENETIC MODULATORS

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Publication number Priority date Publication date Assignee Title
JP2020500526A (ja) * 2016-12-01 2020-01-16 サンガモ セラピューティクス， インコーポレイテッド タウモジュレーターならびにその送達のための方法および組成物

Non-Patent Citations (1)

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BRYAN J. ZEITLER ET AL.: "Sustained Tau Reduction via Zinc Finger Protein Transcription Factors as a potential Next-Generation", ASGCT 20TH ANNUAL MEETING, JPN6024049972, 10 May 2017 (2017-05-10), pages 12, ISSN: 0005665509 *

Also Published As

Publication number Publication date
CN115210251A (zh) 2022-10-18
BR112022014160A2 (pt) 2022-09-13
AU2021209699A1 (en) 2022-07-21
AR121118A1 (es) 2022-04-20
US20230242602A1 (en) 2023-08-03
MX2022008953A (es) 2022-08-15
UY39036A (es) 2021-08-31
WO2021151012A1 (en) 2021-07-29
KR20220131273A (ko) 2022-09-27
TW202134288A (zh) 2021-09-16
EP4093754A1 (en) 2022-11-30
CA3168805A1 (en) 2021-07-29
IL294535A (en) 2022-09-01

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