JP2022551286A - α－シヌクレイン発現を抑制するためのジンクフィンガータンパク質転写因子 - Google Patents

α－シヌクレイン発現を抑制する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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JP2022551286A
JP2022551286A JP2022520779A JP2022520779A JP2022551286A JP 2022551286 A JP2022551286 A JP 2022551286A JP 2022520779 A JP2022520779 A JP 2022520779A JP 2022520779 A JP2022520779 A JP 2022520779A JP 2022551286 A JP2022551286 A JP 2022551286A
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zfp
fusion protein
synuclein
cell
cells
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JP2022551286A5 (https=
Inventor
ハタミ，アサ
ザイトラー，ブライアン
ジャン，レイ
エマニュエル パション，デイビッド
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
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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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—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
    • 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
    • C12N2750/14071—Demonstrated in vivo effect
    • 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)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Wood Science & Technology (AREA)
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  • Immunology (AREA)
  • Plant Pathology (AREA)
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  • Physics & Mathematics (AREA)
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  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022520779A 2019-10-02 2020-10-02 α－シヌクレイン発現を抑制するためのジンクフィンガータンパク質転写因子 Pending JP2022551286A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962909496P 2019-10-02 2019-10-02
US62/909,496 2019-10-02
US202062959153P 2020-01-09 2020-01-09
US62/959,153 2020-01-09
PCT/US2020/054148 WO2021067871A1 (en) 2019-10-02 2020-10-02 Zinc finger protein transcription factors for repressing alpha-synuclein expression

Publications (2)

Publication Number Publication Date
JP2022551286A true JP2022551286A (ja) 2022-12-08
JP2022551286A5 JP2022551286A5 (https=) 2023-10-05

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JP2022520779A Pending JP2022551286A (ja) 2019-10-02 2020-10-02 α－シヌクレイン発現を抑制する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) US20220324928A1 (https=)
EP (1) EP4041754A1 (https=)
JP (1) JP2022551286A (https=)
KR (1) KR20220071248A (https=)
CN (1) CN114466867A (https=)
AU (1) AU2020356962A1 (https=)
CA (1) CA3156258A1 (https=)
IL (1) IL291827A (https=)
TW (2) TW202539725A (https=)
WO (1) WO2021067871A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025006508A2 (en) * 2023-06-26 2025-01-02 Navega Therapeutics, Inc. Compositions and methods for reducing pain and inflammation through epigenetic modulation
WO2025264967A1 (en) * 2024-06-21 2025-12-26 The Johns Hopkins University Agents and/or compositions useful for modulating cis-regulatory elements in synucleinopathies and methods for identifying agents and compositions thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050235369A1 (en) * 2001-03-28 2005-10-20 Yen Choo Gene regulation II
JP2007528367A (ja) * 2003-06-09 2007-10-11 アルナイラム ファーマシューティカルズ インコーポレイテッド 神経変性疾患を治療する方法
WO2019152433A1 (en) * 2018-01-30 2019-08-08 Parkinson's Institute Crispr-based downregulation of alpha-synuclein expression as a novel parkinson's disease therapeutic

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US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
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
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (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.
US7030215B2 (en) * 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
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
WO2002082904A2 (en) * 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
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
US7595306B2 (en) * 2003-06-09 2009-09-29 Alnylam Pharmaceuticals Inc Method of treating neurodegenerative disease
EP1732945B1 (en) * 2004-04-08 2014-12-24 Sangamo BioSciences, Inc. Methods and compositions for modulating cardiac contractility
ES2582091T3 (es) 2005-10-18 2016-09-09 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
HRP20200254T1 (hr) 2010-05-03 2020-05-29 Sangamo Therapeutics, Inc. Pripravci za povezivanje modula cink fingera
WO2012094132A1 (en) * 2011-01-05 2012-07-12 Sangamo Biosciences, Inc. Methods and compositions for gene correction
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
KR102455249B1 (ko) 2016-08-24 2022-10-17 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
IL266862B2 (en) * 2016-12-01 2024-01-01 Sangamo Therapeutics Inc Tau modulators and methods and preparations for their administration
TW202134288A (zh) * 2020-01-22 2021-09-16 美商聖加莫治療股份有限公司 用於抑制tau蛋白表現之鋅指蛋白轉錄因子

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US20050235369A1 (en) * 2001-03-28 2005-10-20 Yen Choo Gene regulation II
JP2007528367A (ja) * 2003-06-09 2007-10-11 アルナイラム ファーマシューティカルズ インコーポレイテッド 神経変性疾患を治療する方法
WO2019152433A1 (en) * 2018-01-30 2019-08-08 Parkinson's Institute Crispr-based downregulation of alpha-synuclein expression as a novel parkinson's disease therapeutic

Also Published As

Publication number Publication date
IL291827A (en) 2022-06-01
US20220324928A1 (en) 2022-10-13
WO2021067871A1 (en) 2021-04-08
TW202539725A (zh) 2025-10-16
TWI888417B (zh) 2025-07-01
EP4041754A1 (en) 2022-08-17
AU2020356962A1 (en) 2022-04-14
KR20220071248A (ko) 2022-05-31
TW202126681A (zh) 2021-07-16
WO2021067871A9 (en) 2021-05-06
CA3156258A1 (en) 2021-04-08
CN114466867A (zh) 2022-05-10

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