AU2010306645B2 - Supercoiled minicircle DNA for gene therapy applications - Google Patents

Supercoiled minicircle DNA for gene therapy applications Download PDF

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Publication number
AU2010306645B2
AU2010306645B2 AU2010306645A AU2010306645A AU2010306645B2 AU 2010306645 B2 AU2010306645 B2 AU 2010306645B2 AU 2010306645 A AU2010306645 A AU 2010306645A AU 2010306645 A AU2010306645 A AU 2010306645A AU 2010306645 B2 AU2010306645 B2 AU 2010306645B2
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Australia
Prior art keywords
minivector
dna
cell
gene
nucleic acid
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Ceased
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AU2010306645A
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AU2010306645A1 (en
Inventor
Erol Bakkalbasi
Daniel James Catanese Jr.
Jonathan Fogg
Brian E. Gilbert
E. Lynn Zechiedrich
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Baylor College of Medicine
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Baylor College of Medicine
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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
    • 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
    • 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
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/90—Vectors containing a transposable element

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • Pulmonology (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2010306645A 2009-10-16 2010-10-15 Supercoiled minicircle DNA for gene therapy applications Ceased AU2010306645B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US25245509P 2009-10-16 2009-10-16
US61/252,455 2009-10-16
PCT/US2010/052920 WO2011047318A2 (en) 2009-10-16 2010-10-15 Supercoiled minicircle dna for gene therapy applications

Publications (2)

Publication Number Publication Date
AU2010306645A1 AU2010306645A1 (en) 2012-06-07
AU2010306645B2 true AU2010306645B2 (en) 2017-03-23

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AU2010306645A Ceased AU2010306645B2 (en) 2009-10-16 2010-10-15 Supercoiled minicircle DNA for gene therapy applications

Country Status (10)

Country Link
US (3) US8460924B2 (enExample)
EP (1) EP2488641B1 (enExample)
JP (2) JP2013507934A (enExample)
KR (1) KR20120094166A (enExample)
AU (1) AU2010306645B2 (enExample)
BR (1) BR112012011576A2 (enExample)
CA (1) CA2777835A1 (enExample)
MX (1) MX2012004447A (enExample)
NZ (1) NZ599998A (enExample)
WO (1) WO2011047318A2 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101510830B1 (ko) 2012-05-11 2015-04-10 가톨릭대학교 산학협력단 생리활성 단백질을 발현하는 미니서클을 이용하여 형질전환된 줄기세포를 통한 치료법
US20150376645A1 (en) * 2012-05-30 2015-12-31 Baylor College Of Medicine Supercoiled minivectors as a tool for dna repair, alteration and replacement
EP2735614A1 (en) * 2012-11-22 2014-05-28 Centre National De La Recherche Scientifique In vitro production of DNA minicircles comprising less than 250 base pairs
JP2016518142A (ja) * 2013-05-10 2016-06-23 サンガモ バイオサイエンシーズ， インコーポレイテッド ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
GB201702500D0 (en) * 2017-02-16 2017-04-05 Univ Sheffield Stable vamp reporter assay
US20180305701A1 (en) 2017-03-14 2018-10-25 Baylor College Of Medicine Targeting minivectors to specific tissue using shape
US20200048716A1 (en) 2017-11-03 2020-02-13 Twister Biotech, Inc Using minivectors to treat ovarian cancer
TW202124722A (zh) 2019-09-18 2021-07-01 美商英特佳樂帝克醫療公司 合成dna載體及其使用方法
US20240093226A1 (en) 2021-09-10 2024-03-21 Baylor College Of Medicine Ultrapure minivectors for gene therapy
US20240100189A1 (en) 2021-09-10 2024-03-28 Twister Biotech, Inc Using minivectors to treat idiopathic pulmonary fibrosis
US20230190955A1 (en) 2021-10-21 2023-06-22 Baylor College Of Medicine Treatment of liver cancer or liver fibrosis

Citations (1)

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WO2004020605A2 (en) * 2002-08-29 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same

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WO1994009127A2 (en) 1992-10-16 1994-04-28 The Government Of The United States Of America As Represented By The Secretary, Department Of Healthand Human Services Supercoiled minicircle dna as as a unitary promoter vector
US6964861B1 (en) * 1998-11-13 2005-11-15 Invitrogen Corporation Enhanced in vitro recombinational cloning of using ribosomal proteins
ATE460472T1 (de) 2000-12-08 2010-03-15 Life Technologies Corp Verfahren und zusammensetzungen zur synthese von nukleinsäuremolekülen unter verwendung multipler erkennungsstellen
GB0108968D0 (en) * 2001-04-10 2001-05-30 Imp College Innovations Ltd Methods
AU2002247848A1 (en) 2001-04-10 2002-10-28 Imperial College Innovations Limited Methods for the production of minicircles
US20030219902A1 (en) 2002-01-31 2003-11-27 Washington State University Research Foundation Methods and vectors for facilitating site-specific recombination
EP1539998A4 (en) 2002-06-05 2007-10-31 Invitrogen Corp METHODS AND COMPOSITIONS FOR SYNTHESIS OF NUCLEIC ACID MOLECULES USING MULTIPLE RECOGNITION SITES
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Publication number Priority date Publication date Assignee Title
WO2004020605A2 (en) * 2002-08-29 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same

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Also Published As

Publication number Publication date
WO2011047318A3 (en) 2011-09-09
MX2012004447A (es) 2012-10-15
WO2011047318A8 (en) 2011-06-09
CA2777835A1 (en) 2011-04-21
KR20120094166A (ko) 2012-08-23
AU2010306645A1 (en) 2012-06-07
US9267150B2 (en) 2016-02-23
NZ599998A (en) 2014-11-28
US8729044B2 (en) 2014-05-20
JP2013507934A (ja) 2013-03-07
WO2011047318A2 (en) 2011-04-21
US20120302625A1 (en) 2012-11-29
EP2488641B1 (en) 2019-01-23
US20130316449A1 (en) 2013-11-28
EP2488641A4 (en) 2013-11-13
US8460924B2 (en) 2013-06-11
US20110160284A1 (en) 2011-06-30
BR112012011576A2 (pt) 2015-09-15
EP2488641A2 (en) 2012-08-22
JP2017046710A (ja) 2017-03-09

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