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 PDFInfo
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- 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
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- 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
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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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
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- 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
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- 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
- C12N2750/14041—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
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- 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
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- 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)
- 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)
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)
| 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)
| 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)
| 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 |
-
2020
- 2020-10-02 AU AU2020356962A patent/AU2020356962A1/en active Pending
- 2020-10-02 US US17/766,076 patent/US20220324928A1/en active Pending
- 2020-10-02 JP JP2022520779A patent/JP2022551286A/ja active Pending
- 2020-10-02 KR KR1020227014231A patent/KR20220071248A/ko active Pending
- 2020-10-02 CA CA3156258A patent/CA3156258A1/en active Pending
- 2020-10-02 EP EP20797308.2A patent/EP4041754A1/en active Pending
- 2020-10-02 CN CN202080069917.0A patent/CN114466867A/zh active Pending
- 2020-10-02 WO PCT/US2020/054148 patent/WO2021067871A1/en not_active Ceased
- 2020-10-05 TW TW114122441A patent/TW202539725A/zh unknown
- 2020-10-05 TW TW109134486A patent/TWI888417B/zh active
-
2022
- 2022-03-30 IL IL291827A patent/IL291827A/en unknown
Patent Citations (5)
| 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)
| 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 |
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