JP2023535025A - Ｄｎａ結合ドメイントランス活性化因子およびその使用 - Google Patents

Ｄｎａ結合ドメイントランス活性化因子およびその使用 Download PDF

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JP2023535025A
JP2023535025A JP2023504286A JP2023504286A JP2023535025A JP 2023535025 A JP2023535025 A JP 2023535025A JP 2023504286 A JP2023504286 A JP 2023504286A JP 2023504286 A JP2023504286 A JP 2023504286A JP 2023535025 A JP2023535025 A JP 2023535025A
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nucleic acid
seq
isolated nucleic
domain
dna binding
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JP2023535025A5 (https=
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ミゲル セナ エステベス，
スコット エー． ウルフ，
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ユニバーシティ オブ マサチューセッツ
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    • C—CHEMISTRY; METALLURGY
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/04—Libraries containing only organic compounds
    • C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
    • C40B40/08—Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
    • C—CHEMISTRY; METALLURGY
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/62—DNA sequences coding for fusion proteins
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/1034—Isolating an individual clone by screening libraries
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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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
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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
    • C12N15/11—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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • 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
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
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    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Physics & Mathematics (AREA)
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  • Crystallography & Structural Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023504286A 2020-07-24 2021-07-23 Ｄｎａ結合ドメイントランス活性化因子およびその使用 Pending JP2023535025A (ja)

Applications Claiming Priority (3)

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US202063056528P 2020-07-24 2020-07-24
US63/056,528 2020-07-24
PCT/US2021/042949 WO2022020706A1 (en) 2020-07-24 2021-07-23 Dna-binding domain transactivators and uses thereof

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JP2023535025A true JP2023535025A (ja) 2023-08-15
JP2023535025A5 JP2023535025A5 (https=) 2024-07-31

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US (1) US20230279405A1 (https=)
EP (1) EP4185303A4 (https=)
JP (1) JP2023535025A (https=)
AR (1) AR123041A1 (https=)
TW (1) TW202221119A (https=)
WO (1) WO2022020706A1 (https=)

Families Citing this family (3)

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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
WO2024238662A2 (en) * 2023-05-17 2024-11-21 University Of Washington Synthetic nuclear localization signals and methods of their use
WO2025250686A1 (en) * 2024-05-28 2025-12-04 The Regents Of The University Of California Methods and systems for parallel spatially resolved single-cell profiling

Citations (4)

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WO2017180915A2 (en) * 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
US20180064827A1 (en) * 2016-09-07 2018-03-08 Sangamo Therapeutics, Inc. Modulation of liver genes
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
WO2019109051A1 (en) * 2017-12-01 2019-06-06 Encoded Therapeutics, Inc. Engineered dna binding proteins

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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
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
MX2019011772A (es) * 2017-04-03 2020-01-09 Encoded Therapeutics Inc Expresion transgenica selectiva de tejidos.
WO2018204764A1 (en) * 2017-05-05 2018-11-08 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
CA3096407A1 (en) * 2018-04-09 2019-10-17 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
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

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2017180915A2 (en) * 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
US20180064827A1 (en) * 2016-09-07 2018-03-08 Sangamo Therapeutics, Inc. Modulation of liver genes
WO2019109051A1 (en) * 2017-12-01 2019-06-06 Encoded Therapeutics, Inc. Engineered dna binding proteins

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WO2022020706A1 (en) 2022-01-27
EP4185303A4 (en) 2024-10-30
TW202221119A (zh) 2022-06-01
EP4185303A1 (en) 2023-05-31
US20230279405A1 (en) 2023-09-07
AR123041A1 (es) 2022-10-26

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