IL294535A - Zinc finger protein transcription factors for repressing tau expression - Google Patents

Zinc finger protein transcription factors for repressing tau expression

Info

Publication number
IL294535A
IL294535A IL294535A IL29453522A IL294535A IL 294535 A IL294535 A IL 294535A IL 294535 A IL294535 A IL 294535A IL 29453522 A IL29453522 A IL 29453522A IL 294535 A IL294535 A IL 294535A
Authority
IL
Israel
Prior art keywords
zfp
fusion protein
dna
cell
domain
Prior art date
Application number
IL294535A
Other languages
English (en)
Hebrew (he)
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 IL294535A publication Critical patent/IL294535A/en

Links

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
    • A61P25/28Drugs 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
    • 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
    • C07K14/4711Alzheimer's disease; Amyloid plaque core protein
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/70Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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

Landscapes

  • 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)
  • 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)
IL294535A 2020-01-22 2021-01-22 Zinc finger protein transcription factors for repressing tau expression IL294535A (en)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
IL294535A true IL294535A (en) 2022-09-01

Family

ID=74661487

Family Applications (1)

Application Number Title Priority Date Filing Date
IL294535A IL294535A (en) 2020-01-22 2021-01-22 Zinc finger protein transcription factors for repressing tau expression

Country Status (14)

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
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
KR20210070344A (ko) 2018-10-02 2021-06-14 상가모 테라퓨틱스, 인코포레이티드 타우 단백질의 조정을 위한 방법 및 조성물
KR20220071248A (ko) * 2019-10-02 2022-05-31 상가모 테라퓨틱스, 인코포레이티드 알파-시누클레인 발현을 억제시키기 위한 아연 핑거 단백질 전사 인자
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
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
ATE524545T1 (de) 1994-08-20 2011-09-15 Gendaq Ltd Verbesserungen bei oder im zusammenhang mit bindeproteinen zur erkennung von dna
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
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
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith 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
US9394531B2 (en) 2008-05-28 2016-07-19 Sangamo Biosciences, Inc. Compositions for linking DNA-binding domains and cleavage domains
PT2566972T (pt) 2010-05-03 2020-03-02 Sangamo Biosciences Inc Composições para a ligação de módulos de dedo de zinco
AU2014312295C1 (en) 2013-08-28 2020-08-13 Sangamo Therapeutics, Inc. 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
EP3411056A4 (en) 2016-02-02 2019-10-02 Sangamo Therapeutics, Inc. COMPOUNDS FOR NETWORKING DNA BINDING DOMAINS AND SPLITTING DOMAINS
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific 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
CA3115158A1 (en) 2018-10-02 2020-04-09 Sangamo Therapeutics, Inc. Engineered genetic modulators
KR20210070344A (ko) 2018-10-02 2021-06-14 상가모 테라퓨틱스, 인코포레이티드 타우 단백질의 조정을 위한 방법 및 조성물

Also Published As

Publication number Publication date
CN115210251A (zh) 2022-10-18
JP2023511908A (ja) 2023-03-23
KR20220131273A (ko) 2022-09-27
TW202134288A (zh) 2021-09-16
EP4093754A1 (en) 2022-11-30
AR121118A1 (es) 2022-04-20
BR112022014160A2 (pt) 2022-09-13
UY39036A (es) 2021-08-31
MX2022008953A (es) 2022-08-15
CA3168805A1 (en) 2021-07-29
WO2021151012A1 (en) 2021-07-29
US20230242602A1 (en) 2023-08-03
AU2021209699A1 (en) 2022-07-21

Similar Documents

Publication Publication Date Title
US20240197919A1 (en) Recombinant aavs for delivery to central nervous system and brain vasculature
IL294535A (en) Zinc finger protein transcription factors for repressing tau expression
AU2024227138A1 (en) Methods and vectors for treating cns disorders
US20220010001A1 (en) Gene therapies for neurodegenerative disease
TW202206446A (zh) 用於治療tdp-43蛋白質病變之組合物及方法
US20230279405A1 (en) Dna-binding domain transactivators and uses thereof
US11795476B2 (en) AAV/UPR-plus virus, UPR-plus fusion protein, genetic treatment method and its use in the treatment of neurodegenerative diseases, such as Parkinson's disease and Huntington's disease, among others
JP2017123840A (ja) グルコーストランスポーター1発現用アデノ随伴ウイルスベクター
EP4410988A1 (en) An aav2-vector variant for targeted transfer of genes
WO2024163823A1 (en) Tissue-specific enhancers for regulating transcription
US20230405149A1 (en) Gene therapies for neurodegenerative disease
JP2023545972A (ja) アルファ-シヌクレイン発現を抑制するための新規ジンクフィンガータンパク質転写因子
WO2025193848A1 (en) Delivery of tau repressors using the blood-brain barrier penetrant capsid
JP2023522852A (ja) Ids遺伝子導入のためのアデノ随伴ウイルス組成物及びその使用方法
TWI883636B (zh) 含有星狀細胞導向啟動子構築體之合成核酸及其使用方法
US12509490B2 (en) AAV capsids for targeting nervous system and uses thereof
AU2024285988A1 (en) Animal model with rapid onset of alzheimer's amyloid beta plaque pathology