AU2015229227B2 - Genomic insulator elements and uses thereof - Google Patents

Genomic insulator elements and uses thereof Download PDF

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Publication number
AU2015229227B2
AU2015229227B2 AU2015229227A AU2015229227A AU2015229227B2 AU 2015229227 B2 AU2015229227 B2 AU 2015229227B2 AU 2015229227 A AU2015229227 A AU 2015229227A AU 2015229227 A AU2015229227 A AU 2015229227A AU 2015229227 B2 AU2015229227 B2 AU 2015229227B2
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seq
vector
ctcf
genomic
viral vector
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AU2015229227A1 (en
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George Stamatoyannopoulos
John STAMATOYANNOPOULOS
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University of Washington
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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)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
AU2015229227A 2014-03-14 2015-03-13 Genomic insulator elements and uses thereof Active AU2015229227B2 (en)

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 (2)

Publication Number Publication Date
AU2015229227A1 AU2015229227A1 (en) 2016-09-29
AU2015229227B2 true AU2015229227B2 (en) 2021-03-18

Family

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Family Applications (1)

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AU2015229227A Active AU2015229227B2 (en) 2014-03-14 2015-03-13 Genomic insulator elements and uses thereof

Country Status (5)

Country Link
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
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
AU2015229227B2 (en) * 2014-03-14 2021-03-18 University Of Washington Genomic insulator elements and uses thereof
EP3620521B1 (en) 2014-09-04 2024-05-29 Memorial Sloan-Kettering Cancer Center Globin gene therapy for treating hemoglobinopathies
EP3380605A1 (en) * 2015-11-24 2018-10-03 GlaxoSmithKline Intellectual Property Development Limited Transient transfection method for retroviral production
WO2018195540A1 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Inducible, tunable, and multiplex human gene regulation using crispr-cpf1
CN111093715A (zh) 2017-09-18 2020-05-01 儿童医院医疗中心 强绝缘子和其在基因递送中的用途
EP3720509A4 (en) 2017-12-06 2021-12-08 Memorial Sloan-Kettering Cancer Center GENE THERAPY WITH A GLOBIN GENE FOR THE TREATMENT OF 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
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
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
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

Citations (2)

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Publication number Priority date Publication date Assignee Title
US20100022006A1 (en) * 2007-02-15 2010-01-28 The Govt. Of The Usa As Represented By The Secreta Gamma satellite insulator sequences and their use in preventing gene silencing
US20120115227A1 (en) * 2009-04-03 2012-05-10 Centre National De La Recherche Scientifique Gene transfer vectors comprising genetic insulator elements and methods to identify genetic insulator elements

Family Cites Families (14)

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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
CA2377929A1 (en) 1999-06-30 2001-01-11 Adam C. Bell 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
US20100144543A1 (en) * 2008-05-01 2010-06-10 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
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
US9566329B2 (en) * 2012-04-06 2017-02-14 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Live, attenuated rubella vector to express vaccine antigens
AU2015229227B2 (en) * 2014-03-14 2021-03-18 University Of Washington Genomic insulator elements and uses thereof
EP3620521B1 (en) 2014-09-04 2024-05-29 Memorial Sloan-Kettering Cancer Center Globin gene therapy for treating hemoglobinopathies

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100022006A1 (en) * 2007-02-15 2010-01-28 The Govt. Of The Usa As Represented By The Secreta Gamma satellite insulator sequences and their use in preventing gene silencing
US20120115227A1 (en) * 2009-04-03 2012-05-10 Centre National De La Recherche Scientifique Gene transfer vectors comprising genetic insulator elements and methods to identify genetic insulator elements

Also Published As

Publication number Publication date
US20240182924A1 (en) 2024-06-06
US20200263202A1 (en) 2020-08-20
AU2015229227A1 (en) 2016-09-29
EP3117004A1 (en) 2017-01-18
WO2015138852A1 (en) 2015-09-17
JP2017510293A (ja) 2017-04-13
US10590433B2 (en) 2020-03-17
US20170175136A1 (en) 2017-06-22
US11788101B2 (en) 2023-10-17
EP3117004A4 (en) 2017-12-06
JP6737714B2 (ja) 2020-08-12

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