CN114466867A - 用于抑制α-突触核蛋白表达的锌指蛋白转录因子 - Google Patents

用于抑制α-突触核蛋白表达的锌指蛋白转录因子 Download PDF

Info

Publication number
CN114466867A
CN114466867A CN202080069917.0A CN202080069917A CN114466867A CN 114466867 A CN114466867 A CN 114466867A CN 202080069917 A CN202080069917 A CN 202080069917A CN 114466867 A CN114466867 A CN 114466867A
Authority
CN
China
Prior art keywords
arg
thr
lys
ser
ala
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN202080069917.0A
Other languages
English (en)
Chinese (zh)
Inventor
A.哈塔米
B.蔡特勒
L.张
D.E.帕肖恩
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sangamo Therapeutics Inc
Original Assignee
Sangamo Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of CN114466867A publication Critical patent/CN114466867A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • 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
    • 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
    • C12N2750/14041Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/14071Demonstrated in vivo effect
    • 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

Landscapes

  • 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)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Psychiatry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Toxicology (AREA)
  • Psychology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202080069917.0A 2019-10-02 2020-10-02 用于抑制α-突触核蛋白表达的锌指蛋白转录因子 Pending CN114466867A (zh)

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 (1)

Publication Number Publication Date
CN114466867A true CN114466867A (zh) 2022-05-10

Family

ID=73014653

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080069917.0A Pending CN114466867A (zh) 2019-10-02 2020-10-02 用于抑制α-突触核蛋白表达的锌指蛋白转录因子

Country Status (10)

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 (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050186591A1 (en) * 2003-06-09 2005-08-25 Alnylam Pharmaceuticals Method of treating neurodegenerative disease
US20070161595A1 (en) * 2003-06-09 2007-07-12 Mayo Foundation For Medical Education And Research Method of treating neurodegenerative disease
US20180153921A1 (en) * 2016-12-01 2018-06-07 Sangamo Therapeutics, Inc. Tau modulators and methods and compositions for delivery thereof
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
CN115210251A (zh) * 2020-01-22 2022-10-18 桑格摩生物治疗股份有限公司 用于阻遏tau表达的锌指蛋白转录因子

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20050235369A1 (en) * 2001-03-28 2005-10-20 Yen Choo Gene regulation II
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
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

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050186591A1 (en) * 2003-06-09 2005-08-25 Alnylam Pharmaceuticals Method of treating neurodegenerative disease
US20070161595A1 (en) * 2003-06-09 2007-07-12 Mayo Foundation For Medical Education And Research Method of treating neurodegenerative disease
US20180153921A1 (en) * 2016-12-01 2018-06-07 Sangamo Therapeutics, Inc. Tau modulators and methods and compositions for delivery thereof
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
CN115210251A (zh) * 2020-01-22 2022-10-18 桑格摩生物治疗股份有限公司 用于阻遏tau表达的锌指蛋白转录因子

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
DAVID E. PASCHON等: "Diversifying the structure of zinc finger nucleases for high-precision genome editing", NATURE COMMUNICATIONS, 8 March 2019 (2019-03-08) *
毛超;陶永光;: "基因组编辑技术研究新进展", 生命的化学, no. 01, 15 February 2015 (2015-02-15), pages 96 - 104 *

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
JP2022551286A (ja) 2022-12-08
TW202126681A (zh) 2021-07-16
WO2021067871A9 (en) 2021-05-06
CA3156258A1 (en) 2021-04-08

Similar Documents

Publication Publication Date Title
CN113766921B (zh) 染色体9开放阅读框72基因表达的调节物及其用途
US20230242602A1 (en) Zinc finger protein transcription factors for repressing tau expression
JP7824873B2 (ja) プリオン病の治療のためのジンクフィンガータンパク質転写因子
CN114466867A (zh) 用于抑制α-突触核蛋白表达的锌指蛋白转录因子
TW202229320A (zh) 用於抑制α-突觸核蛋白(SYNUCLEIN)表現之新穎鋅指蛋白轉錄因子
RU2828216C2 (ru) Модуляторы экспрессии гена открытой рамки считывания 72 хромосомы 9 и их применения
WO2025194009A1 (en) Zinc finger proteins for treating prion disease
TW202547548A (zh) 使用血腦屏障穿透性蛋白殼之tau抑制子遞送

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination