JP6737714B2 - ゲノムインスレーターエレメントおよびその使用 - Google Patents

ゲノムインスレーターエレメントおよびその使用 Download PDF

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JP6737714B2
JP6737714B2 JP2016575630A JP2016575630A JP6737714B2 JP 6737714 B2 JP6737714 B2 JP 6737714B2 JP 2016575630 A JP2016575630 A JP 2016575630A JP 2016575630 A JP2016575630 A JP 2016575630A JP 6737714 B2 JP6737714 B2 JP 6737714B2
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vector
genomic
sequence
ctcf
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JP2017510293A5 (enExample
JP2017510293A (ja
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ジョージ スタマトヤンノプロス
ジョージ スタマトヤンノプロス
ジョン スタマトヤンノプロス
ジョン スタマトヤンノプロス
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University of Washington
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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
    • C12N15/63—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
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2016575630A 2014-03-14 2015-03-13 ゲノムインスレーターエレメントおよびその使用 Active JP6737714B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461953419P 2014-03-14 2014-03-14
US61/953,419 2014-03-14
US201462068226P 2014-10-24 2014-10-24
US62/068,226 2014-10-24
PCT/US2015/020369 WO2015138852A1 (en) 2014-03-14 2015-03-13 Genomic insulator elements and uses thereof

Publications (3)

Publication Number Publication Date
JP2017510293A JP2017510293A (ja) 2017-04-13
JP2017510293A5 JP2017510293A5 (enExample) 2018-04-26
JP6737714B2 true JP6737714B2 (ja) 2020-08-12

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JP2016575630A Active JP6737714B2 (ja) 2014-03-14 2015-03-13 ゲノムインスレーターエレメントおよびその使用

Country Status (5)

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US (3) US10590433B2 (enExample)
EP (1) EP3117004A4 (enExample)
JP (1) JP6737714B2 (enExample)
AU (1) AU2015229227B2 (enExample)
WO (1) WO2015138852A1 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105408483A (zh) 2013-03-15 2016-03-16 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
EP3117004A4 (en) * 2014-03-14 2017-12-06 University of Washington Genomic insulator elements and uses thereof
BR112017004349A2 (pt) * 2014-09-04 2017-12-05 Memorial Sloan Kettering Cancer Center terapia com gene de globina para o tratamento de hemoglobinopatias
KR102067352B1 (ko) * 2015-11-24 2020-01-16 글락소스미스클라인 인털렉츄얼 프로퍼티 디벨로프먼트 리미티드 레트로바이러스 생산을 위한 일시적인 형질감염 방법
EP3612204A4 (en) 2017-04-21 2021-01-27 The General Hospital Corporation INDUCTIBLE, TUNING AND MULTIPLEX HUMAN GENE REGULATION USING CRISPR-CPF1
US11970707B2 (en) 2017-09-18 2024-04-30 Children's Hospital Medical Center Strong insulator and uses thereof in gene delivery
EP3720509A4 (en) 2017-12-06 2021-12-08 Memorial Sloan-Kettering Cancer Center GENE THERAPY WITH A GLOBIN GENE FOR THE TREATMENT OF HEMOGLOBINOPATHIES
CA3100726A1 (en) 2018-05-17 2019-11-21 The General Hospital Corporation Ccctc-binding factor variants
WO2020040046A1 (ja) * 2018-08-21 2020-02-27 学校法人慶應義塾 エンハンサーブロッカー及びその使用
CN110747233A (zh) * 2019-08-21 2020-02-04 常州市第二人民医院 含一种绝缘子的慢病毒稳定表达载体及其构建方法
CN110760543B (zh) * 2019-08-21 2021-12-10 常州市第二人民医院 一种运用绝缘子组合构建的慢病毒多启动子稳定表达载体及构建方法
US12702720B2 (en) 2020-05-14 2026-08-11 St. Jude Children's Research Hospital, Inc. Genomic insulator element exhibiting enhancer blocking activities in lymphocytes and uses thereof
WO2024201066A1 (en) 2023-03-31 2024-10-03 Dawn Therapeutics Limited Novel vector
EP4790141A1 (en) * 2023-10-08 2026-08-12 Shanghai Qilu Pharmaceutical Research and Development Centre Ltd. Truncated insulator and use thereof in transient expression of recombinant protein
EP4545644A1 (en) * 2023-10-24 2025-04-30 Ospedale San Raffaele S.r.l. Polynucleotides
WO2025235425A1 (en) * 2024-05-06 2025-11-13 Research Institute At Nationwide Children's Hospital Improved proviral plasmids
WO2026064752A1 (en) * 2024-09-22 2026-03-26 Newberry Skyler Compositions and methods for in vivo genetic engineering of lymphocyte precursors

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US4897268A (en) 1987-08-03 1990-01-30 Southern Research Institute Drug delivery system and method of making the same
US5610053A (en) * 1993-04-07 1997-03-11 The United States Of America As Represented By The Department Of Health And Human Services DNA sequence which acts as a chromatin insulator element to protect expressed genes from cis-acting regulatory sequences in mammalian cells
US5972643A (en) 1994-06-17 1999-10-26 Fred Hutchinson Cancer Research Center Isolated polynucleotide molecules encoding CTCF, a CCCTC-binding factor
AU776752B2 (en) 1999-06-30 2004-09-23 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The DNA binding protein and sequence as insulators having specific enhancer blocking activity for regulation of gene expression
WO2002088346A2 (en) 2001-05-01 2002-11-07 National Research Council Of Canada A system for inducible expression in eukaryotic cells
US20050283854A1 (en) 2003-05-23 2005-12-22 Anton Krumm Recombinant vectors for use in position-independent transgene expression within chromatin
WO2008101216A2 (en) 2007-02-15 2008-08-21 The Govt. Of The Usa As Represented By The Secretary Of The Dept Of Health And Human Services Gamma satellite insulator sequences and their use in preventing gene silencing
WO2009134420A2 (en) 2008-05-01 2009-11-05 The Salk Institute For Biological Studies Epigenetic silencing of tumor suppressor genes
US20110294873A1 (en) * 2008-10-23 2011-12-01 Université de Lausanne Gene Transfer Vectors Comprising At Least One Isolated DNA Molecule Having Insulator Or Boundary Properties And Methods To Identify The Same
WO2010108143A2 (en) * 2009-03-20 2010-09-23 University Of Washington Global mapping of protein-dna interaction by digital genomic footprinting
EP2414524B1 (en) * 2009-04-03 2017-08-23 Centre National De La Recherche Scientifique Gene transfer vectors comprising genetic insulator elements and methods to identify genetic insulator elements
US20110294114A1 (en) 2009-12-04 2011-12-01 Cincinnati Children's Hospital Medical Center Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
WO2013152352A1 (en) * 2012-04-06 2013-10-10 The United States Of America, As Represented By The Secretary, Department Of Health And Human Servic Live, attenuated rubella vector to express vaccine antigens
EP3117004A4 (en) * 2014-03-14 2017-12-06 University of Washington Genomic insulator elements and uses thereof
BR112017004349A2 (pt) 2014-09-04 2017-12-05 Memorial Sloan Kettering Cancer Center terapia com gene de globina para o tratamento de hemoglobinopatias

Also Published As

Publication number Publication date
EP3117004A4 (en) 2017-12-06
AU2015229227A1 (en) 2016-09-29
AU2015229227B2 (en) 2021-03-18
JP2017510293A (ja) 2017-04-13
WO2015138852A1 (en) 2015-09-17
US20200263202A1 (en) 2020-08-20
US11788101B2 (en) 2023-10-17
EP3117004A1 (en) 2017-01-18
US20240182924A1 (en) 2024-06-06
US10590433B2 (en) 2020-03-17
US20170175136A1 (en) 2017-06-22

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