CN113710693B - Dna结合结构域反式激活因子及其用途 - Google Patents
Dna结合结构域反式激活因子及其用途 Download PDFInfo
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- CN113710693B CN113710693B CN202080021751.5A CN202080021751A CN113710693B CN 113710693 B CN113710693 B CN 113710693B CN 202080021751 A CN202080021751 A CN 202080021751A CN 113710693 B CN113710693 B CN 113710693B
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- A61P25/08—Antiepileptics; Anticonvulsants
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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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
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
- 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
-
- 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
- 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
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- 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
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Pain & Pain Management (AREA)
- Epidemiology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962810005P | 2019-02-25 | 2019-02-25 | |
| US62/810,005 | 2019-02-25 | ||
| PCT/US2020/019546 WO2020176426A1 (en) | 2019-02-25 | 2020-02-24 | Dna-binding domain transactivators and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN113710693A CN113710693A (zh) | 2021-11-26 |
| CN113710693B true CN113710693B (zh) | 2025-07-15 |
Family
ID=72238705
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202080021751.5A Active CN113710693B (zh) | 2019-02-25 | 2020-02-24 | Dna结合结构域反式激活因子及其用途 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US12583902B2 (https=) |
| EP (1) | EP3931213A4 (https=) |
| JP (2) | JP7624728B2 (https=) |
| KR (1) | KR20210132109A (https=) |
| CN (1) | CN113710693B (https=) |
| AU (2) | AU2020228028A1 (https=) |
| BR (1) | BR112021016294A2 (https=) |
| CA (1) | CA3129757A1 (https=) |
| CL (1) | CL2021002226A1 (https=) |
| CO (1) | CO2021011722A2 (https=) |
| IL (1) | IL285687A (https=) |
| MX (1) | MX2021010169A (https=) |
| SG (1) | SG11202108568YA (https=) |
| WO (1) | WO2020176426A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7624728B2 (ja) | 2019-02-25 | 2025-01-31 | ユニバーシティ オブ マサチューセッツ | Dna結合ドメイントランスアクチベーター及びその使用 |
| WO2022020706A1 (en) * | 2020-07-24 | 2022-01-27 | University Of Massachusetts | Dna-binding domain transactivators and uses thereof |
| WO2023042104A1 (en) * | 2021-09-16 | 2023-03-23 | Novartis Ag | Novel transcription factors |
| JP2024545923A (ja) * | 2021-12-30 | 2024-12-13 | リーゲル セラピューティクス,インコーポレイティッド | ナトリウム電位依存性チャネルアルファサブユニット1の発現を調節するための組成物、及びその使用 |
| KR20250016657A (ko) * | 2023-07-21 | 2025-02-04 | 한국화학연구원 | dxCas9 및 CRP 유도체를 포함하는, 표적 유전자 발현 조절 시스템 및 이의 제조방법 |
Family Cites Families (49)
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| US5549910A (en) | 1989-03-31 | 1996-08-27 | The Regents Of The University Of California | Preparation of liposome and lipid complex compositions |
| US5252334A (en) | 1989-09-08 | 1993-10-12 | Cygnus Therapeutic Systems | Solid matrix system for transdermal drug delivery |
| JP3218637B2 (ja) | 1990-07-26 | 2001-10-15 | 大正製薬株式会社 | 安定なリポソーム水懸濁液 |
| JP2958076B2 (ja) | 1990-08-27 | 1999-10-06 | 株式会社ビタミン研究所 | 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法 |
| US5399363A (en) | 1991-01-25 | 1995-03-21 | Eastman Kodak Company | Surface modified anticancer nanoparticles |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| US5741516A (en) | 1994-06-20 | 1998-04-21 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| US5795587A (en) | 1995-01-23 | 1998-08-18 | University Of Pittsburgh | Stable lipid-comprising drug delivery complexes and methods for their production |
| US5697899A (en) | 1995-02-07 | 1997-12-16 | Gensia | Feedback controlled drug delivery system |
| IE80468B1 (en) | 1995-04-04 | 1998-07-29 | Elan Corp Plc | Controlled release biodegradable nanoparticles containing insulin |
| US5738868A (en) | 1995-07-18 | 1998-04-14 | Lipogenics Ltd. | Liposome compositions and kits therefor |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| US5656016A (en) | 1996-03-18 | 1997-08-12 | Abbott Laboratories | Sonophoretic drug delivery system |
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| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| ES2341926T3 (es) | 1998-03-02 | 2010-06-29 | Massachusetts Institute Of Technology | Poliproteinas con dedos de cinc que tienen enlazadores mejorados. |
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| US20030082561A1 (en) | 2000-07-21 | 2003-05-01 | Takashi Sera | Zinc finger domain recognition code and uses thereof |
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| US20060182736A1 (en) | 2003-06-10 | 2006-08-17 | Kim Jin-Soo | Transducible dna-binding proteins |
| WO2007061759A1 (en) | 2005-11-18 | 2007-05-31 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Delayed expression vectors |
| AU2010273220B2 (en) | 2009-07-13 | 2015-10-15 | President And Fellows Of Harvard College | Bifunctional stapled polypeptides and uses thereof |
| US9567573B2 (en) * | 2010-04-26 | 2017-02-14 | Sangamo Biosciences, Inc. | Genome editing of a Rosa locus using nucleases |
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| JP2018513681A (ja) | 2015-03-31 | 2018-05-31 | エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. | 細胞または生物のゲノムへのDNA配列の標的化組み込みのためのCas9レトロウイルスインテグラーゼおよびCas9レコンビナーゼ系 |
| JP6853257B2 (ja) | 2015-09-23 | 2021-03-31 | サンガモ セラピューティクス, インコーポレイテッド | Httリプレッサー及びその使用 |
| WO2017053729A1 (en) | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
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| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
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| JP2018088888A (ja) * | 2016-12-06 | 2018-06-14 | 国立研究開発法人理化学研究所 | Scn1a遺伝子の発現増強法 |
| US11730828B2 (en) | 2017-02-07 | 2023-08-22 | The Regents Of The University Of California | Gene therapy for haploinsufficiency |
| MX2019011772A (es) | 2017-04-03 | 2020-01-09 | Encoded Therapeutics Inc | Expresion transgenica selectiva de tejidos. |
| EP3717505A4 (en) | 2017-12-01 | 2021-12-01 | Encoded Therapeutics, Inc. | GENENICALLY MODIFIED DNA-BINDING PROTEINS |
| CA3096407A1 (en) | 2018-04-09 | 2019-10-17 | Allen Institute | Rescuing voltage-gated sodium channel function in inhibitory neurons |
| WO2019224864A1 (ja) | 2018-05-21 | 2019-11-28 | 国立研究開発法人理化学研究所 | Scn1a遺伝子の発現増強法とそれによるドラベ症候群の治療法 |
| WO2020097121A1 (en) | 2018-11-05 | 2020-05-14 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons |
| JP7624728B2 (ja) | 2019-02-25 | 2025-01-31 | ユニバーシティ オブ マサチューセッツ | Dna結合ドメイントランスアクチベーター及びその使用 |
| PL3976798T3 (pl) | 2019-05-29 | 2026-04-07 | Encoded Therapeutics, Inc. | Kompozycje i sposoby selektywnej regulacji genów |
| WO2022020706A1 (en) | 2020-07-24 | 2022-01-27 | University Of Massachusetts | Dna-binding domain transactivators and uses thereof |
-
2020
- 2020-02-24 JP JP2021549631A patent/JP7624728B2/ja active Active
- 2020-02-24 BR BR112021016294-1A patent/BR112021016294A2/pt not_active IP Right Cessation
- 2020-02-24 CA CA3129757A patent/CA3129757A1/en active Pending
- 2020-02-24 US US17/433,269 patent/US12583902B2/en active Active
- 2020-02-24 MX MX2021010169A patent/MX2021010169A/es unknown
- 2020-02-24 EP EP20762996.5A patent/EP3931213A4/en active Pending
- 2020-02-24 CN CN202080021751.5A patent/CN113710693B/zh active Active
- 2020-02-24 WO PCT/US2020/019546 patent/WO2020176426A1/en not_active Ceased
- 2020-02-24 SG SG11202108568YA patent/SG11202108568YA/en unknown
- 2020-02-24 KR KR1020217030158A patent/KR20210132109A/ko not_active Ceased
- 2020-02-24 AU AU2020228028A patent/AU2020228028A1/en not_active Abandoned
-
2021
- 2021-08-17 IL IL285687A patent/IL285687A/en unknown
- 2021-08-23 CL CL2021002226A patent/CL2021002226A1/es unknown
- 2021-09-06 CO CONC2021/0011722A patent/CO2021011722A2/es unknown
-
2024
- 2024-10-18 JP JP2024184019A patent/JP2025003531A/ja active Pending
-
2025
- 2025-09-22 AU AU2025234289A patent/AU2025234289A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| BR112021016294A2 (pt) | 2021-10-13 |
| US20220185862A1 (en) | 2022-06-16 |
| CA3129757A1 (en) | 2020-09-03 |
| CO2021011722A2 (es) | 2021-12-10 |
| CL2021002226A1 (es) | 2022-04-29 |
| US12583902B2 (en) | 2026-03-24 |
| AU2025234289A1 (en) | 2025-10-16 |
| CN113710693A (zh) | 2021-11-26 |
| WO2020176426A1 (en) | 2020-09-03 |
| EP3931213A1 (en) | 2022-01-05 |
| IL285687A (en) | 2021-10-31 |
| JP2025003531A (ja) | 2025-01-09 |
| JP7624728B2 (ja) | 2025-01-31 |
| EP3931213A4 (en) | 2022-12-14 |
| KR20210132109A (ko) | 2021-11-03 |
| MX2021010169A (es) | 2021-12-10 |
| SG11202108568YA (en) | 2021-09-29 |
| JP2022521432A (ja) | 2022-04-07 |
| AU2020228028A1 (en) | 2021-09-30 |
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