TW202134288A - 用於抑制tau蛋白表現之鋅指蛋白轉錄因子 - Google Patents

用於抑制tau蛋白表現之鋅指蛋白轉錄因子 Download PDF

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TW202134288A
TW202134288A TW110102602A TW110102602A TW202134288A TW 202134288 A TW202134288 A TW 202134288A TW 110102602 A TW110102602 A TW 110102602A TW 110102602 A TW110102602 A TW 110102602A TW 202134288 A TW202134288 A TW 202134288A
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zfp
fusion protein
sequence
protein
domain
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TW110102602A
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布萊恩 齊特勒
阿薩 哈塔米
雷 張
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美商聖加莫治療股份有限公司
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    • 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
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    • 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)
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  • 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)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
TW110102602A 2020-01-22 2021-01-22 用於抑制tau蛋白表現之鋅指蛋白轉錄因子 TW202134288A (zh)

Applications Claiming Priority (2)

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US202062964501P 2020-01-22 2020-01-22
US62/964,501 2020-01-22

Publications (1)

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TW202134288A true TW202134288A (zh) 2021-09-16

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

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110214184B (zh) 2016-12-01 2024-07-02 桑格摩生物治疗股份有限公司 τ蛋白调节剂以及用于其递送的方法和组合物
IL326834A (en) 2018-10-02 2026-04-01 Sangamo Therapeutics Inc Artificial zinc finger protein transcription factors repressing mapt expression for the prevention and/or treatment of tauopathies
WO2021067871A1 (en) * 2019-10-02 2021-04-08 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

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
JP4012243B2 (ja) 1994-01-18 2007-11-21 ザ スクリップス リサーチ インスティチュート 亜鉛フィンガータンパク質誘導体およびそのための方法
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
CA2196419C (en) 1994-08-20 2007-08-21 Yen Choo Improvements in or relating to 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
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
WO2001053480A1 (en) 2000-01-24 2001-07-26 Sangamo Biosciences, Inc. Nucleic acid binding polypeptides characterized by flexible linkers connected nucleic acid binding modules
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
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
CA2725773C (en) 2008-05-28 2017-12-05 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
EP2566972B1 (en) 2010-05-03 2020-01-15 Sangamo Therapeutics, Inc. Compositions for linking zinc finger modules
CA2920899C (en) 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
EP3929279A1 (en) 2014-03-18 2021-12-29 Sangamo Therapeutics, Inc. Methods and compositions for regulation of zinc finger protein expression
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
JP7012650B2 (ja) 2016-02-02 2022-01-28 サンガモ セラピューティクス， インコーポレイテッド Ｄｎａ結合ドメインと切断ドメインとを連結するための組成物
IL264792B2 (en) 2016-08-24 2023-10-01 Sangamo Therapeutics Inc Engineered target-specific zinc-finger nucleases
CN110214184B (zh) * 2016-12-01 2024-07-02 桑格摩生物治疗股份有限公司 τ蛋白调节剂以及用于其递送的方法和组合物
KR20210069692A (ko) 2018-10-02 2021-06-11 상가모 테라퓨틱스, 인코포레이티드 조작된 유전자 조정제
IL326834A (en) 2018-10-02 2026-04-01 Sangamo Therapeutics Inc Artificial zinc finger protein transcription factors repressing mapt expression for the prevention and/or treatment of tauopathies

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UY39036A (es) 2021-08-31
BR112022014160A2 (pt) 2022-09-13
JP2023511908A (ja) 2023-03-23
MX2022008953A (es) 2022-08-15
IL294535A (en) 2022-09-01
WO2021151012A1 (en) 2021-07-29
CN115210251A (zh) 2022-10-18
KR20220131273A (ko) 2022-09-27
EP4093754A1 (en) 2022-11-30
AR121118A1 (es) 2022-04-20
US20230242602A1 (en) 2023-08-03
AU2021209699A1 (en) 2022-07-21
CA3168805A1 (en) 2021-07-29

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