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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ユニヴァーシティ オブ ワシントン
ユニヴァーシティ オブ ワシントン
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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)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (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)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Epidemiology (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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)

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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

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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 (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
US10590433B2 (en) * 2014-03-14 2020-03-17 University Of Washington Genomic insulator elements and uses thereof
KR102500531B1 (ko) * 2014-09-04 2023-02-17 메모리얼 슬로안 케터링 캔서 센터 헤모글로빈병증 치료용 글로빈 유전자 치료법
FR3044017B1 (fr) * 2015-11-24 2019-05-03 Glaxosmithkline Intellectual Property Development Limited Procede de transfection transitoire pour la production retrovirale
CA3059208A1 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Inducible, tunable, and multiplex human gene regulation using crispr-cpf1
WO2019056015A2 (en) * 2017-09-18 2019-03-21 Children's Hospital Medical Center STRONG ISOLATOR AND ITS USES IN GENE ADMINISTRATION
AU2018378683B2 (en) 2017-12-06 2025-10-09 Memorial Sloan-Kettering Cancer Center Globin gene therapy for treating hemoglobinopathies
EP3793584A4 (en) 2018-05-17 2022-10-26 The General Hospital Corporation CCCTC BINDING FACTOR VARIATIONS
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
US20230181762A1 (en) * 2020-05-14 2023-06-15 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
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

Family Cites Families (16)

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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
JP2003504016A (ja) 1999-06-30 2003-02-04 アメリカ合衆国 遺伝子発現の制御のための特異的エンハンサー遮断活性を有する絶縁体としてのｄｎａ結合タンパク質および配列
AU2002257420A1 (en) 2001-05-01 2002-11-11 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
US9018011B2 (en) * 2007-02-15 2015-04-28 The United States As Represented By The Secretary Of The Department Of Health And Human Services Gamma satellite insulator sequences and their use in preventing gene silencing
US20100144543A1 (en) * 2008-05-01 2010-06-10 The Salk Institute For Biological Studies Epigenetic silencing of tumor suppressor genes
EP2344641A2 (en) * 2008-10-23 2011-07-20 Université de Lausanne Gene transfer vectors comprising at least one isolated dna molecule having insulator and or boundary properties and methods to identify the same
US20120178641A1 (en) * 2009-03-20 2012-07-12 Stamatoyannopoulos John A Global mapping of protein-dna interaction by digital genomic footprinting
US8828718B2 (en) 2009-04-03 2014-09-09 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
US10590433B2 (en) * 2014-03-14 2020-03-17 University Of Washington Genomic insulator elements and uses thereof
KR102500531B1 (ko) 2014-09-04 2023-02-17 메모리얼 슬로안 케터링 캔서 센터 헤모글로빈병증 치료용 글로빈 유전자 치료법

Also Published As

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

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